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Gene Therapy for Dystrophic EB: Extension Study Results Reported
and no new safety signals were identified.
The results were presented by Amy S. Paller, MD, during a late-breaking session at the annual meeting of the American Academy of Dermatology.
In May 2023, beremagene geperpavec, marketed as Vyjuvek (formerly known as B-VEC) was approved by the US Food and Drug Administration (FDA) for the treatment of wounds in patients 6 months of age and older with DEB, a rare genetic blistering disorder caused by COL7A1 gene variants. The therapy uses a nonreplicating herpes simplex virus type 1 (HSV-1) vector to deliver the COL7A1 gene directly to skin cells, restoring the COL7 protein fibrils that stabilize skin structure. It is designed to be used repetitively, to heal a single wound, or on more than one wound.
In the pivotal study of patients with DEB, the gene therapy, delivered in a topical gel, was administered once a week for 6 months to one wound and placebo was applied to another wound for each participant. The proportion of wounds treated with beremagene geperpavec that healed was significantly higher than among placebo-treated wounds at 3 and 6 months (68% vs. 23% at 3 months, P = .003) and 65% vs. 26% at 6 months (P = .012), with no serious adverse events related to treatment.
The prospective, open label, uncontrolled extension study included 24 patients from the phase 3 study and 23 treatment-naive patients from five US sites. Their mean age was 16 years (range, 6 months to 46 years).
Of the 47 patients, 29 (62%) were on treatment for more than 1 year (the longest was about 2 years), and the mean duration of treatment was 475 days; 5 patients withdrew from the study for reasons not related to treatment.
Their types of adverse events (AEs) were similar to those seen in the phase 3 study and were consistent with what would be expected in patients with DEB, said Dr. Paller, professor and chair of dermatology, Northwestern University, Chicago. One patient experienced two wound hemorrhages that were possibly related to treatment, but there were no treatment-related AEs, no deaths or treatment discontinuations because of an AE, and no serious AEs thought to be related to treatment.
Wounds that were evaluated in the phase 3 study showed “a high durability of closure with continued treatment,” according to Dr. Paller. There were enough data on 19 of the 24 patients who had been in the phase 3 trial to evaluate wound closure, defined as “complete wound closure based on comparison to the exact wound area selected at baseline” at the beginning of the phase 3 study.
In the extension study, wound closure rates were almost 90% at baseline, 84.2% at 3 months, 61.1% at 6 months, 82.4% at 9 months, and 62.5% at 12 months, which was comparable to the rates observed in the third (86.4%) and sixth (73.7%) months of the phase 3 study, Dr. Paller said.
Patient-reported outcomes indicated that quality of life and satisfaction with treatment were preserved with continued treatment.The extension study was terminated in July 2023, after FDA approval, when patients could be transitioned to the commercially available treatment.Dr. Paller disclosed being an investigator (funds to institution) for multiple pharmaceutical companies, including the manufacturer of beremagene geperpavec, Krystal Biotech, which funded the study.
and no new safety signals were identified.
The results were presented by Amy S. Paller, MD, during a late-breaking session at the annual meeting of the American Academy of Dermatology.
In May 2023, beremagene geperpavec, marketed as Vyjuvek (formerly known as B-VEC) was approved by the US Food and Drug Administration (FDA) for the treatment of wounds in patients 6 months of age and older with DEB, a rare genetic blistering disorder caused by COL7A1 gene variants. The therapy uses a nonreplicating herpes simplex virus type 1 (HSV-1) vector to deliver the COL7A1 gene directly to skin cells, restoring the COL7 protein fibrils that stabilize skin structure. It is designed to be used repetitively, to heal a single wound, or on more than one wound.
In the pivotal study of patients with DEB, the gene therapy, delivered in a topical gel, was administered once a week for 6 months to one wound and placebo was applied to another wound for each participant. The proportion of wounds treated with beremagene geperpavec that healed was significantly higher than among placebo-treated wounds at 3 and 6 months (68% vs. 23% at 3 months, P = .003) and 65% vs. 26% at 6 months (P = .012), with no serious adverse events related to treatment.
The prospective, open label, uncontrolled extension study included 24 patients from the phase 3 study and 23 treatment-naive patients from five US sites. Their mean age was 16 years (range, 6 months to 46 years).
Of the 47 patients, 29 (62%) were on treatment for more than 1 year (the longest was about 2 years), and the mean duration of treatment was 475 days; 5 patients withdrew from the study for reasons not related to treatment.
Their types of adverse events (AEs) were similar to those seen in the phase 3 study and were consistent with what would be expected in patients with DEB, said Dr. Paller, professor and chair of dermatology, Northwestern University, Chicago. One patient experienced two wound hemorrhages that were possibly related to treatment, but there were no treatment-related AEs, no deaths or treatment discontinuations because of an AE, and no serious AEs thought to be related to treatment.
Wounds that were evaluated in the phase 3 study showed “a high durability of closure with continued treatment,” according to Dr. Paller. There were enough data on 19 of the 24 patients who had been in the phase 3 trial to evaluate wound closure, defined as “complete wound closure based on comparison to the exact wound area selected at baseline” at the beginning of the phase 3 study.
In the extension study, wound closure rates were almost 90% at baseline, 84.2% at 3 months, 61.1% at 6 months, 82.4% at 9 months, and 62.5% at 12 months, which was comparable to the rates observed in the third (86.4%) and sixth (73.7%) months of the phase 3 study, Dr. Paller said.
Patient-reported outcomes indicated that quality of life and satisfaction with treatment were preserved with continued treatment.The extension study was terminated in July 2023, after FDA approval, when patients could be transitioned to the commercially available treatment.Dr. Paller disclosed being an investigator (funds to institution) for multiple pharmaceutical companies, including the manufacturer of beremagene geperpavec, Krystal Biotech, which funded the study.
and no new safety signals were identified.
The results were presented by Amy S. Paller, MD, during a late-breaking session at the annual meeting of the American Academy of Dermatology.
In May 2023, beremagene geperpavec, marketed as Vyjuvek (formerly known as B-VEC) was approved by the US Food and Drug Administration (FDA) for the treatment of wounds in patients 6 months of age and older with DEB, a rare genetic blistering disorder caused by COL7A1 gene variants. The therapy uses a nonreplicating herpes simplex virus type 1 (HSV-1) vector to deliver the COL7A1 gene directly to skin cells, restoring the COL7 protein fibrils that stabilize skin structure. It is designed to be used repetitively, to heal a single wound, or on more than one wound.
In the pivotal study of patients with DEB, the gene therapy, delivered in a topical gel, was administered once a week for 6 months to one wound and placebo was applied to another wound for each participant. The proportion of wounds treated with beremagene geperpavec that healed was significantly higher than among placebo-treated wounds at 3 and 6 months (68% vs. 23% at 3 months, P = .003) and 65% vs. 26% at 6 months (P = .012), with no serious adverse events related to treatment.
The prospective, open label, uncontrolled extension study included 24 patients from the phase 3 study and 23 treatment-naive patients from five US sites. Their mean age was 16 years (range, 6 months to 46 years).
Of the 47 patients, 29 (62%) were on treatment for more than 1 year (the longest was about 2 years), and the mean duration of treatment was 475 days; 5 patients withdrew from the study for reasons not related to treatment.
Their types of adverse events (AEs) were similar to those seen in the phase 3 study and were consistent with what would be expected in patients with DEB, said Dr. Paller, professor and chair of dermatology, Northwestern University, Chicago. One patient experienced two wound hemorrhages that were possibly related to treatment, but there were no treatment-related AEs, no deaths or treatment discontinuations because of an AE, and no serious AEs thought to be related to treatment.
Wounds that were evaluated in the phase 3 study showed “a high durability of closure with continued treatment,” according to Dr. Paller. There were enough data on 19 of the 24 patients who had been in the phase 3 trial to evaluate wound closure, defined as “complete wound closure based on comparison to the exact wound area selected at baseline” at the beginning of the phase 3 study.
In the extension study, wound closure rates were almost 90% at baseline, 84.2% at 3 months, 61.1% at 6 months, 82.4% at 9 months, and 62.5% at 12 months, which was comparable to the rates observed in the third (86.4%) and sixth (73.7%) months of the phase 3 study, Dr. Paller said.
Patient-reported outcomes indicated that quality of life and satisfaction with treatment were preserved with continued treatment.The extension study was terminated in July 2023, after FDA approval, when patients could be transitioned to the commercially available treatment.Dr. Paller disclosed being an investigator (funds to institution) for multiple pharmaceutical companies, including the manufacturer of beremagene geperpavec, Krystal Biotech, which funded the study.
FROM AAD 2024
Positive Phase 2 Results Reported for TYK2 Inhibitor for Patients With Moderate to Severe Psoriasis
, in a phase 2 study presented during a late breaker presentation at the annual meeting of the American Academy of Dermatology.
ESK-001 is a highly-selective TYK2 inhibitor that “reduces signaling through several cytokine receptors including receptors for interleukin (IL)-12, IL-23, and interferon (IFN)-alpha,” according to Alumis, the company developing ESK-001.
The STRIDE trial enrolled 228 adults (mean age, 48 years) with a mean PASI score of 17.8 at baseline, randomized to one of five doses of ESK-001, or placebo. Nearly 70% of those enrolled were men, 82.5% were White, nearly 6% were Asian, and about 4% were Black.
The proportion of patients achieving at least a PASI-75 at 12 weeks, the primary endpoint, ranged from 19.4% among those on the lower dose (10 mg a day) to 56.4% among those on 20 mg twice a day and those on 40 mg once a day, and 64.1% among those on 40 mg twice a day, the highest dose evaluated, compared with 0% of those on placebo.
In addition, 38.5% and 15.4% of those on the highest dose achieved a PASI 90 and PASI 100, respectively, at 12 weeks, according to results presented at the meeting by Kim A. Papp, MD.
Improvements in static Physician’s Global Assessment (sPGA) scores, a secondary endpoint, at 12 weeks were also observed; including 59% of those on the highest dose (40 mg twice a day) achieving an sPGA of 0/1 at 12 weeks, according to Dr. Papp, president of Probity Medical Research, in Waterloo, Ontario, and a study investigator.
Treatment was “generally safe and well tolerated” at all doses, and most treatment-emergent adverse events were “mild to moderate” and self-limited, he said.
The most frequent treatment emergent adverse events included headache and nasopharyngitis (also reported in the placebo group), and upper respiratory tract infections. No cases of treatment-related serious adverse events were reported, and there were no cases of MACE (major adverse cardiovascular events), serious infections, thromboses related to treatment, or lab or ECG findings of concern.
In an ongoing open label extension study, 165 patients on 40 mg once a day and those on 40 mg twice a day are continuing to be evaluated, and at week 16, have continued to show significant efficacy, with a favorable risk-benefit profile to date, according to Dr. Papp.
ESK-001 is also being evaluated in a phase 2b study of patients with systemic lupus erythematosus, and a phase 2 proof-of-concept study of patients with non-infectious uveitis, according to Alumis.
In addition to being an investigator for Alumis, Dr. Papp disclosed serving as an adviser, consultant, investigator, and/or speaker for multiple other pharmaceutical companies.
, in a phase 2 study presented during a late breaker presentation at the annual meeting of the American Academy of Dermatology.
ESK-001 is a highly-selective TYK2 inhibitor that “reduces signaling through several cytokine receptors including receptors for interleukin (IL)-12, IL-23, and interferon (IFN)-alpha,” according to Alumis, the company developing ESK-001.
The STRIDE trial enrolled 228 adults (mean age, 48 years) with a mean PASI score of 17.8 at baseline, randomized to one of five doses of ESK-001, or placebo. Nearly 70% of those enrolled were men, 82.5% were White, nearly 6% were Asian, and about 4% were Black.
The proportion of patients achieving at least a PASI-75 at 12 weeks, the primary endpoint, ranged from 19.4% among those on the lower dose (10 mg a day) to 56.4% among those on 20 mg twice a day and those on 40 mg once a day, and 64.1% among those on 40 mg twice a day, the highest dose evaluated, compared with 0% of those on placebo.
In addition, 38.5% and 15.4% of those on the highest dose achieved a PASI 90 and PASI 100, respectively, at 12 weeks, according to results presented at the meeting by Kim A. Papp, MD.
Improvements in static Physician’s Global Assessment (sPGA) scores, a secondary endpoint, at 12 weeks were also observed; including 59% of those on the highest dose (40 mg twice a day) achieving an sPGA of 0/1 at 12 weeks, according to Dr. Papp, president of Probity Medical Research, in Waterloo, Ontario, and a study investigator.
Treatment was “generally safe and well tolerated” at all doses, and most treatment-emergent adverse events were “mild to moderate” and self-limited, he said.
The most frequent treatment emergent adverse events included headache and nasopharyngitis (also reported in the placebo group), and upper respiratory tract infections. No cases of treatment-related serious adverse events were reported, and there were no cases of MACE (major adverse cardiovascular events), serious infections, thromboses related to treatment, or lab or ECG findings of concern.
In an ongoing open label extension study, 165 patients on 40 mg once a day and those on 40 mg twice a day are continuing to be evaluated, and at week 16, have continued to show significant efficacy, with a favorable risk-benefit profile to date, according to Dr. Papp.
ESK-001 is also being evaluated in a phase 2b study of patients with systemic lupus erythematosus, and a phase 2 proof-of-concept study of patients with non-infectious uveitis, according to Alumis.
In addition to being an investigator for Alumis, Dr. Papp disclosed serving as an adviser, consultant, investigator, and/or speaker for multiple other pharmaceutical companies.
, in a phase 2 study presented during a late breaker presentation at the annual meeting of the American Academy of Dermatology.
ESK-001 is a highly-selective TYK2 inhibitor that “reduces signaling through several cytokine receptors including receptors for interleukin (IL)-12, IL-23, and interferon (IFN)-alpha,” according to Alumis, the company developing ESK-001.
The STRIDE trial enrolled 228 adults (mean age, 48 years) with a mean PASI score of 17.8 at baseline, randomized to one of five doses of ESK-001, or placebo. Nearly 70% of those enrolled were men, 82.5% were White, nearly 6% were Asian, and about 4% were Black.
The proportion of patients achieving at least a PASI-75 at 12 weeks, the primary endpoint, ranged from 19.4% among those on the lower dose (10 mg a day) to 56.4% among those on 20 mg twice a day and those on 40 mg once a day, and 64.1% among those on 40 mg twice a day, the highest dose evaluated, compared with 0% of those on placebo.
In addition, 38.5% and 15.4% of those on the highest dose achieved a PASI 90 and PASI 100, respectively, at 12 weeks, according to results presented at the meeting by Kim A. Papp, MD.
Improvements in static Physician’s Global Assessment (sPGA) scores, a secondary endpoint, at 12 weeks were also observed; including 59% of those on the highest dose (40 mg twice a day) achieving an sPGA of 0/1 at 12 weeks, according to Dr. Papp, president of Probity Medical Research, in Waterloo, Ontario, and a study investigator.
Treatment was “generally safe and well tolerated” at all doses, and most treatment-emergent adverse events were “mild to moderate” and self-limited, he said.
The most frequent treatment emergent adverse events included headache and nasopharyngitis (also reported in the placebo group), and upper respiratory tract infections. No cases of treatment-related serious adverse events were reported, and there were no cases of MACE (major adverse cardiovascular events), serious infections, thromboses related to treatment, or lab or ECG findings of concern.
In an ongoing open label extension study, 165 patients on 40 mg once a day and those on 40 mg twice a day are continuing to be evaluated, and at week 16, have continued to show significant efficacy, with a favorable risk-benefit profile to date, according to Dr. Papp.
ESK-001 is also being evaluated in a phase 2b study of patients with systemic lupus erythematosus, and a phase 2 proof-of-concept study of patients with non-infectious uveitis, according to Alumis.
In addition to being an investigator for Alumis, Dr. Papp disclosed serving as an adviser, consultant, investigator, and/or speaker for multiple other pharmaceutical companies.
FROM AAD 2024
FDA warns about off-label use of laparoscopic device for aesthetic procedures
The .
The device is cleared by the FDA for “general use of cutting, coagulation, and ablation of soft tissue during open and laparoscopic surgical procedures” but it “has not been determined to be safe or effective for any procedure intended to improve the appearance of the skin,” according to the March 14 statement from the FDA. The statement adds that the agency has received reports describing “serious and potentially life-threatening adverse events with use of this device for certain aesthetic procedures,” including some that have required treatment in an intensive care unit. The statement does not mention whether any cases were fatal.
Adverse events that have been reported include second- and third-degree burns, infections, changes in skin color, scars, nerve damage, “significant bleeding,” and “air or gas accumulation under the skin, in body cavities, and in blood vessels.”
Manufactured by Apyx medical, the device includes a hand piece and generator and uses radiofrequency energy and helium to generate plasma, which is used to “cut, coagulate ... and eliminate soft tissue with heat during surgery,” according to the FDA.
The FDA is advising health care providers not to use the device for dermal resurfacing or skin contraction “alone or in combination with liposuction.”
The statement also advises consumers who are considering an aesthetic skin treatment with this device to consult their health care providers regarding its use – and if they have any problems or are concerned after being treated with this device, to “seek care from a licensed health care provider.”
The FDA is working with Apyx to evaluate information about the use of the device for aesthetic skin procedures and to inform consumers and health care providers about the warning.
Health care providers and consumers should report problems or complications associated with the Renuvion/J-Plasma device to the FDA’s MedWatch program.
A version of this article first appeared on Medscape.com.
The .
The device is cleared by the FDA for “general use of cutting, coagulation, and ablation of soft tissue during open and laparoscopic surgical procedures” but it “has not been determined to be safe or effective for any procedure intended to improve the appearance of the skin,” according to the March 14 statement from the FDA. The statement adds that the agency has received reports describing “serious and potentially life-threatening adverse events with use of this device for certain aesthetic procedures,” including some that have required treatment in an intensive care unit. The statement does not mention whether any cases were fatal.
Adverse events that have been reported include second- and third-degree burns, infections, changes in skin color, scars, nerve damage, “significant bleeding,” and “air or gas accumulation under the skin, in body cavities, and in blood vessels.”
Manufactured by Apyx medical, the device includes a hand piece and generator and uses radiofrequency energy and helium to generate plasma, which is used to “cut, coagulate ... and eliminate soft tissue with heat during surgery,” according to the FDA.
The FDA is advising health care providers not to use the device for dermal resurfacing or skin contraction “alone or in combination with liposuction.”
The statement also advises consumers who are considering an aesthetic skin treatment with this device to consult their health care providers regarding its use – and if they have any problems or are concerned after being treated with this device, to “seek care from a licensed health care provider.”
The FDA is working with Apyx to evaluate information about the use of the device for aesthetic skin procedures and to inform consumers and health care providers about the warning.
Health care providers and consumers should report problems or complications associated with the Renuvion/J-Plasma device to the FDA’s MedWatch program.
A version of this article first appeared on Medscape.com.
The .
The device is cleared by the FDA for “general use of cutting, coagulation, and ablation of soft tissue during open and laparoscopic surgical procedures” but it “has not been determined to be safe or effective for any procedure intended to improve the appearance of the skin,” according to the March 14 statement from the FDA. The statement adds that the agency has received reports describing “serious and potentially life-threatening adverse events with use of this device for certain aesthetic procedures,” including some that have required treatment in an intensive care unit. The statement does not mention whether any cases were fatal.
Adverse events that have been reported include second- and third-degree burns, infections, changes in skin color, scars, nerve damage, “significant bleeding,” and “air or gas accumulation under the skin, in body cavities, and in blood vessels.”
Manufactured by Apyx medical, the device includes a hand piece and generator and uses radiofrequency energy and helium to generate plasma, which is used to “cut, coagulate ... and eliminate soft tissue with heat during surgery,” according to the FDA.
The FDA is advising health care providers not to use the device for dermal resurfacing or skin contraction “alone or in combination with liposuction.”
The statement also advises consumers who are considering an aesthetic skin treatment with this device to consult their health care providers regarding its use – and if they have any problems or are concerned after being treated with this device, to “seek care from a licensed health care provider.”
The FDA is working with Apyx to evaluate information about the use of the device for aesthetic skin procedures and to inform consumers and health care providers about the warning.
Health care providers and consumers should report problems or complications associated with the Renuvion/J-Plasma device to the FDA’s MedWatch program.
A version of this article first appeared on Medscape.com.
FDA updates status of iPLEDGE access problems
The, one month after a modified program was launched, the Food and Drug Administration announced on Jan. 14.
The IPMG has “created a new tool within the system to help resolve account access for some user groups without using the call center. This tool is intended to allow prescribers and designees to send login links directly to their patients’ desired email address through the Manage Patients page of the iPLEDGE REMS portal,” the FDA statement said.
“Prescribers can also send login links to their designees still having difficulty accessing their iPLEDGE account,” and users should check their emails for messages from iPLEDGE, including spam folders, the FDA advises. The iPLEDGE strategy is designed to prevent fetal exposure to isotretinoin, which is highly teratogenic.
Days after the new, gender-neutral approach to the isotretinoin risk mitigation program was launched on Dec. 13, the FDA convened an emergency meeting with representatives from the American Academy of Dermatology Association (AADA) to discuss the problematic rollout of the program, which was described as disastrous, chaotic, and a failure, with dermatologists on Twitter and elsewhere expressing anger and frustration over not being able to access the program or reach the call center.
A statement by the FDA on Dec. 23 followed, urging manufacturers to develop solutions for the website and to work with the AADA and pharmacy organizations to find solutions that would minimize treatment interruptions during the transition.
The modified REMS, launched on Dec. 13, is designed to make it more inclusive for transgender patients prescribed isotretinoin. Instead of three risk categories (females of reproductive potential, females not of reproductive potential, and males), patients who are prescribed isotretinoin for acne are assigned to one of two risk categories: those who can get pregnant and those who cannot get pregnant.
In the Jan. 14 statement, the FDA notes that the agency is continuing to work with the IPMG regarding the problems clinicians, pharmacists, and patients have had with accessing iPLEDGE over the last month.
“Although there has been progress, there is a significant amount of work still to be done,” the FDA acknowledged. “While we consider potential steps within the scope of FDA’s authorities, we will continue to meet with the IPMG for updates on the status of the problems with the iPLEDGE REMS and their progress towards having the system work as intended for all users.”
The, one month after a modified program was launched, the Food and Drug Administration announced on Jan. 14.
The IPMG has “created a new tool within the system to help resolve account access for some user groups without using the call center. This tool is intended to allow prescribers and designees to send login links directly to their patients’ desired email address through the Manage Patients page of the iPLEDGE REMS portal,” the FDA statement said.
“Prescribers can also send login links to their designees still having difficulty accessing their iPLEDGE account,” and users should check their emails for messages from iPLEDGE, including spam folders, the FDA advises. The iPLEDGE strategy is designed to prevent fetal exposure to isotretinoin, which is highly teratogenic.
Days after the new, gender-neutral approach to the isotretinoin risk mitigation program was launched on Dec. 13, the FDA convened an emergency meeting with representatives from the American Academy of Dermatology Association (AADA) to discuss the problematic rollout of the program, which was described as disastrous, chaotic, and a failure, with dermatologists on Twitter and elsewhere expressing anger and frustration over not being able to access the program or reach the call center.
A statement by the FDA on Dec. 23 followed, urging manufacturers to develop solutions for the website and to work with the AADA and pharmacy organizations to find solutions that would minimize treatment interruptions during the transition.
The modified REMS, launched on Dec. 13, is designed to make it more inclusive for transgender patients prescribed isotretinoin. Instead of three risk categories (females of reproductive potential, females not of reproductive potential, and males), patients who are prescribed isotretinoin for acne are assigned to one of two risk categories: those who can get pregnant and those who cannot get pregnant.
In the Jan. 14 statement, the FDA notes that the agency is continuing to work with the IPMG regarding the problems clinicians, pharmacists, and patients have had with accessing iPLEDGE over the last month.
“Although there has been progress, there is a significant amount of work still to be done,” the FDA acknowledged. “While we consider potential steps within the scope of FDA’s authorities, we will continue to meet with the IPMG for updates on the status of the problems with the iPLEDGE REMS and their progress towards having the system work as intended for all users.”
The, one month after a modified program was launched, the Food and Drug Administration announced on Jan. 14.
The IPMG has “created a new tool within the system to help resolve account access for some user groups without using the call center. This tool is intended to allow prescribers and designees to send login links directly to their patients’ desired email address through the Manage Patients page of the iPLEDGE REMS portal,” the FDA statement said.
“Prescribers can also send login links to their designees still having difficulty accessing their iPLEDGE account,” and users should check their emails for messages from iPLEDGE, including spam folders, the FDA advises. The iPLEDGE strategy is designed to prevent fetal exposure to isotretinoin, which is highly teratogenic.
Days after the new, gender-neutral approach to the isotretinoin risk mitigation program was launched on Dec. 13, the FDA convened an emergency meeting with representatives from the American Academy of Dermatology Association (AADA) to discuss the problematic rollout of the program, which was described as disastrous, chaotic, and a failure, with dermatologists on Twitter and elsewhere expressing anger and frustration over not being able to access the program or reach the call center.
A statement by the FDA on Dec. 23 followed, urging manufacturers to develop solutions for the website and to work with the AADA and pharmacy organizations to find solutions that would minimize treatment interruptions during the transition.
The modified REMS, launched on Dec. 13, is designed to make it more inclusive for transgender patients prescribed isotretinoin. Instead of three risk categories (females of reproductive potential, females not of reproductive potential, and males), patients who are prescribed isotretinoin for acne are assigned to one of two risk categories: those who can get pregnant and those who cannot get pregnant.
In the Jan. 14 statement, the FDA notes that the agency is continuing to work with the IPMG regarding the problems clinicians, pharmacists, and patients have had with accessing iPLEDGE over the last month.
“Although there has been progress, there is a significant amount of work still to be done,” the FDA acknowledged. “While we consider potential steps within the scope of FDA’s authorities, we will continue to meet with the IPMG for updates on the status of the problems with the iPLEDGE REMS and their progress towards having the system work as intended for all users.”
Abrocitinib approved for atopic dermatitis in Europe
who are candidates for systemic therapy, the manufacturer announced.
Approval by the European Commission was based on the results of studies that include four phase 3 clinical trials (JADE MONO-1, JADE-MONO-2, JADE COMPARE, JADE REGIMEN) and an ongoing open-label extension study (JADE EXTEND) in over 2,800 patients, according to the Pfizer press release announcing the approval. The approved doses are 100 and 200 mg a day; a 50-mg dose was approved for patients with moderate and severe renal impairment and “ certain patients receiving treatment with inhibitors of cytochrome P450 (CYP) 2C19,” the release said.
The approval follows a positive opinion by the Committee for Medicinal Products for Human Use of the European Medicines Agency supporting marketing authorization for treating AD, issued in October. It will be marketed as Cibinqo.
Abrocitinib is under review at the Food and Drug Administration. It was approved earlier in 2021 for treating AD in the United Kingdom, Japan, and Korea.
emechcatie@mdedge.com
who are candidates for systemic therapy, the manufacturer announced.
Approval by the European Commission was based on the results of studies that include four phase 3 clinical trials (JADE MONO-1, JADE-MONO-2, JADE COMPARE, JADE REGIMEN) and an ongoing open-label extension study (JADE EXTEND) in over 2,800 patients, according to the Pfizer press release announcing the approval. The approved doses are 100 and 200 mg a day; a 50-mg dose was approved for patients with moderate and severe renal impairment and “ certain patients receiving treatment with inhibitors of cytochrome P450 (CYP) 2C19,” the release said.
The approval follows a positive opinion by the Committee for Medicinal Products for Human Use of the European Medicines Agency supporting marketing authorization for treating AD, issued in October. It will be marketed as Cibinqo.
Abrocitinib is under review at the Food and Drug Administration. It was approved earlier in 2021 for treating AD in the United Kingdom, Japan, and Korea.
emechcatie@mdedge.com
who are candidates for systemic therapy, the manufacturer announced.
Approval by the European Commission was based on the results of studies that include four phase 3 clinical trials (JADE MONO-1, JADE-MONO-2, JADE COMPARE, JADE REGIMEN) and an ongoing open-label extension study (JADE EXTEND) in over 2,800 patients, according to the Pfizer press release announcing the approval. The approved doses are 100 and 200 mg a day; a 50-mg dose was approved for patients with moderate and severe renal impairment and “ certain patients receiving treatment with inhibitors of cytochrome P450 (CYP) 2C19,” the release said.
The approval follows a positive opinion by the Committee for Medicinal Products for Human Use of the European Medicines Agency supporting marketing authorization for treating AD, issued in October. It will be marketed as Cibinqo.
Abrocitinib is under review at the Food and Drug Administration. It was approved earlier in 2021 for treating AD in the United Kingdom, Japan, and Korea.
emechcatie@mdedge.com
European agency supports marketing of abrocitinib for atopic dermatitis
The
The full indication is for the treatment of moderate to severe AD in adults who are candidates for systemic therapy, according to a summary of the opinion, made on Oct. 14. It will be available as 50-mg, 100-mg, and 200-mg tablets, will be marketed under the name Cibinqo, and “should be prescribed by physicians experienced in the treatment of atopic dermatitis,” the statement said.
“The benefits of Cibinqo are its ability to improve the skin condition as measured by improvements in the Investigator’s Global Assessment 0/1 and Eczema Area and Severity Index 75 response and to reduce itching in patients with atopic dermatitis,” according to the opinion. The most common side effects of abrocitinib are nausea, headache, acne, herpes simplex, increased blood creatine phosphokinase, vomiting, dizziness, and upper abdominal pain, the statement said, and infections are the most serious.
Abrocitinib was first approved for AD in the United Kingdom and in Japan in September, and is under review at the Food and Drug Administration for this indication. The first JAK inhibitor approved for AD in the United States is topical ruxolitinib (Opzelura), approved in September, for the short-term, noncontinuous chronic treatment of mild to moderate AD in nonimmunocompromised patients aged 12 years and older whose disease is not adequately controlled with topical prescription treatments, “or when those therapies are not advisable.”
The
The full indication is for the treatment of moderate to severe AD in adults who are candidates for systemic therapy, according to a summary of the opinion, made on Oct. 14. It will be available as 50-mg, 100-mg, and 200-mg tablets, will be marketed under the name Cibinqo, and “should be prescribed by physicians experienced in the treatment of atopic dermatitis,” the statement said.
“The benefits of Cibinqo are its ability to improve the skin condition as measured by improvements in the Investigator’s Global Assessment 0/1 and Eczema Area and Severity Index 75 response and to reduce itching in patients with atopic dermatitis,” according to the opinion. The most common side effects of abrocitinib are nausea, headache, acne, herpes simplex, increased blood creatine phosphokinase, vomiting, dizziness, and upper abdominal pain, the statement said, and infections are the most serious.
Abrocitinib was first approved for AD in the United Kingdom and in Japan in September, and is under review at the Food and Drug Administration for this indication. The first JAK inhibitor approved for AD in the United States is topical ruxolitinib (Opzelura), approved in September, for the short-term, noncontinuous chronic treatment of mild to moderate AD in nonimmunocompromised patients aged 12 years and older whose disease is not adequately controlled with topical prescription treatments, “or when those therapies are not advisable.”
The
The full indication is for the treatment of moderate to severe AD in adults who are candidates for systemic therapy, according to a summary of the opinion, made on Oct. 14. It will be available as 50-mg, 100-mg, and 200-mg tablets, will be marketed under the name Cibinqo, and “should be prescribed by physicians experienced in the treatment of atopic dermatitis,” the statement said.
“The benefits of Cibinqo are its ability to improve the skin condition as measured by improvements in the Investigator’s Global Assessment 0/1 and Eczema Area and Severity Index 75 response and to reduce itching in patients with atopic dermatitis,” according to the opinion. The most common side effects of abrocitinib are nausea, headache, acne, herpes simplex, increased blood creatine phosphokinase, vomiting, dizziness, and upper abdominal pain, the statement said, and infections are the most serious.
Abrocitinib was first approved for AD in the United Kingdom and in Japan in September, and is under review at the Food and Drug Administration for this indication. The first JAK inhibitor approved for AD in the United States is topical ruxolitinib (Opzelura), approved in September, for the short-term, noncontinuous chronic treatment of mild to moderate AD in nonimmunocompromised patients aged 12 years and older whose disease is not adequately controlled with topical prescription treatments, “or when those therapies are not advisable.”
FDA issues warning about use of dermal fillers with needle-free devices
.
Specifically, the warning advises consumers and health care professionals “not to use needle-free devices such as hyaluron pens for injection of hyaluronic acid (HA) or other lip and facial fillers, collectively and commonly referred to as dermal fillers or fillers.”
According to the statement, the agency “is aware of serious injuries and in some cases, permanent harm to the skin, lips, or eyes with the use of needle-free devices for injection of fillers.”
Needle-free devices and lip and facial fillers for use with these devices are being sold directly to consumers online, and are promoted on social media “to increase lip volume, improve the appearance of wrinkles, change the shape of the nose, and other similar procedures,” according to the FDA warning.
The FDA points out that FDA-approved dermal fillers are for prescription use only, and should be administered only by licensed health care professionals using a syringe with a needle or cannula, and advises consumers not to buy or use lip or facial fillers sold directly to the public.
These products may be contaminated with infectious agents or chemicals. Moreover, “needle-free injection devices for aesthetic purposes do not provide enough control over where the injected product is placed,” the statement adds. In addition to infections, other risks include bleeding and bruising, formation of lumps, allergic reactions, blockage of a blood vessel (which can result in necrosis, blindness, or stroke), and transmission of diseases from sharing devices.
The FDA’s recommendations for health care providers include not using any aesthetic fillers with a needle-free device, and not using approved dermal fillers in such devices.
The American Society for Dermatologic Surgery Association (ASDSA) commended the FDA on the safety communication in a statement issued on October 11. In February, the ASDSA issued an alert about children using hyaluron pens to self-inject hyaluronic filler into the epidermal and upper dermal skin layers.
“I am pleased that the FDA has taken notice of this disturbing new trend, especially that of children using these devices on social media,” ASDSA president Mathew Avram, MD, JD, director of the Dermatology Laser and Cosmetic Center, at Massachusetts General Hospital, Boston, said in the statement. “The complexity of facial anatomy requires in-depth knowledge and expertise, and patients should always have medical procedures done by a physician who also has knowledge of adverse events,” he added, urging consumers to see a board-certified dermatologist before undergoing any cosmetic procedure.
In response to a query, an FDA spokesperson did not have an estimate of the number of reports of these adverse events.
People who have problems or are concerned about having had a filler injected with a needle-free device should contact a licensed health care provider. Consumers and health care professionals should report adverse events related to injection of fillers with a needle-free device to the FDA’s MedWatch program. In addition to MedWatch, adverse events can also be reported to the Cutaneous Procedures Adverse Events Reporting (CAPER) Registry, established earlier this year by the ASDSA with the department of dermatology at Northwestern University, Chicago.
*This story was updated on October 12.
.
Specifically, the warning advises consumers and health care professionals “not to use needle-free devices such as hyaluron pens for injection of hyaluronic acid (HA) or other lip and facial fillers, collectively and commonly referred to as dermal fillers or fillers.”
According to the statement, the agency “is aware of serious injuries and in some cases, permanent harm to the skin, lips, or eyes with the use of needle-free devices for injection of fillers.”
Needle-free devices and lip and facial fillers for use with these devices are being sold directly to consumers online, and are promoted on social media “to increase lip volume, improve the appearance of wrinkles, change the shape of the nose, and other similar procedures,” according to the FDA warning.
The FDA points out that FDA-approved dermal fillers are for prescription use only, and should be administered only by licensed health care professionals using a syringe with a needle or cannula, and advises consumers not to buy or use lip or facial fillers sold directly to the public.
These products may be contaminated with infectious agents or chemicals. Moreover, “needle-free injection devices for aesthetic purposes do not provide enough control over where the injected product is placed,” the statement adds. In addition to infections, other risks include bleeding and bruising, formation of lumps, allergic reactions, blockage of a blood vessel (which can result in necrosis, blindness, or stroke), and transmission of diseases from sharing devices.
The FDA’s recommendations for health care providers include not using any aesthetic fillers with a needle-free device, and not using approved dermal fillers in such devices.
The American Society for Dermatologic Surgery Association (ASDSA) commended the FDA on the safety communication in a statement issued on October 11. In February, the ASDSA issued an alert about children using hyaluron pens to self-inject hyaluronic filler into the epidermal and upper dermal skin layers.
“I am pleased that the FDA has taken notice of this disturbing new trend, especially that of children using these devices on social media,” ASDSA president Mathew Avram, MD, JD, director of the Dermatology Laser and Cosmetic Center, at Massachusetts General Hospital, Boston, said in the statement. “The complexity of facial anatomy requires in-depth knowledge and expertise, and patients should always have medical procedures done by a physician who also has knowledge of adverse events,” he added, urging consumers to see a board-certified dermatologist before undergoing any cosmetic procedure.
In response to a query, an FDA spokesperson did not have an estimate of the number of reports of these adverse events.
People who have problems or are concerned about having had a filler injected with a needle-free device should contact a licensed health care provider. Consumers and health care professionals should report adverse events related to injection of fillers with a needle-free device to the FDA’s MedWatch program. In addition to MedWatch, adverse events can also be reported to the Cutaneous Procedures Adverse Events Reporting (CAPER) Registry, established earlier this year by the ASDSA with the department of dermatology at Northwestern University, Chicago.
*This story was updated on October 12.
.
Specifically, the warning advises consumers and health care professionals “not to use needle-free devices such as hyaluron pens for injection of hyaluronic acid (HA) or other lip and facial fillers, collectively and commonly referred to as dermal fillers or fillers.”
According to the statement, the agency “is aware of serious injuries and in some cases, permanent harm to the skin, lips, or eyes with the use of needle-free devices for injection of fillers.”
Needle-free devices and lip and facial fillers for use with these devices are being sold directly to consumers online, and are promoted on social media “to increase lip volume, improve the appearance of wrinkles, change the shape of the nose, and other similar procedures,” according to the FDA warning.
The FDA points out that FDA-approved dermal fillers are for prescription use only, and should be administered only by licensed health care professionals using a syringe with a needle or cannula, and advises consumers not to buy or use lip or facial fillers sold directly to the public.
These products may be contaminated with infectious agents or chemicals. Moreover, “needle-free injection devices for aesthetic purposes do not provide enough control over where the injected product is placed,” the statement adds. In addition to infections, other risks include bleeding and bruising, formation of lumps, allergic reactions, blockage of a blood vessel (which can result in necrosis, blindness, or stroke), and transmission of diseases from sharing devices.
The FDA’s recommendations for health care providers include not using any aesthetic fillers with a needle-free device, and not using approved dermal fillers in such devices.
The American Society for Dermatologic Surgery Association (ASDSA) commended the FDA on the safety communication in a statement issued on October 11. In February, the ASDSA issued an alert about children using hyaluron pens to self-inject hyaluronic filler into the epidermal and upper dermal skin layers.
“I am pleased that the FDA has taken notice of this disturbing new trend, especially that of children using these devices on social media,” ASDSA president Mathew Avram, MD, JD, director of the Dermatology Laser and Cosmetic Center, at Massachusetts General Hospital, Boston, said in the statement. “The complexity of facial anatomy requires in-depth knowledge and expertise, and patients should always have medical procedures done by a physician who also has knowledge of adverse events,” he added, urging consumers to see a board-certified dermatologist before undergoing any cosmetic procedure.
In response to a query, an FDA spokesperson did not have an estimate of the number of reports of these adverse events.
People who have problems or are concerned about having had a filler injected with a needle-free device should contact a licensed health care provider. Consumers and health care professionals should report adverse events related to injection of fillers with a needle-free device to the FDA’s MedWatch program. In addition to MedWatch, adverse events can also be reported to the Cutaneous Procedures Adverse Events Reporting (CAPER) Registry, established earlier this year by the ASDSA with the department of dermatology at Northwestern University, Chicago.
*This story was updated on October 12.
Dermatology News welcomes new advisory board member
at University Hospital Saint-Louis in Paris, in the inflammatory diseases outpatient clinic, where he treats patients with severe psoriasis and other inflammatory chronic skin diseases.
He is a member of several dermatology specialty organizations, including the Société Française de Dermatologie, the European Academy of Dermatology, as well as the American Academy of Dermatology. His research interests are in immunology and inflammatory diseases; he also has a passion for art and history.
at University Hospital Saint-Louis in Paris, in the inflammatory diseases outpatient clinic, where he treats patients with severe psoriasis and other inflammatory chronic skin diseases.
He is a member of several dermatology specialty organizations, including the Société Française de Dermatologie, the European Academy of Dermatology, as well as the American Academy of Dermatology. His research interests are in immunology and inflammatory diseases; he also has a passion for art and history.
at University Hospital Saint-Louis in Paris, in the inflammatory diseases outpatient clinic, where he treats patients with severe psoriasis and other inflammatory chronic skin diseases.
He is a member of several dermatology specialty organizations, including the Société Française de Dermatologie, the European Academy of Dermatology, as well as the American Academy of Dermatology. His research interests are in immunology and inflammatory diseases; he also has a passion for art and history.
Rosacea: Target treatment to pathogenic pathway
LAHAINA, HAWAII – The pathophysiology of rosacea is complicated, which is why “we try to target our treatments to various areas in this pathogenic pathway” to achieve optimal results, according to Linda Stein Gold, MD, director of dermatology research at the Henry Ford Health System in Detroit.
For example, in a patient with papules and pustules, a topical or oral anti-inflammatory agent is needed “to calm that down.” If background erythema is present, separate from papules and pustules, use a topical alpha-adrenergic agonist, she advised. For telangiectasias, consider a device-based treatment, and for a phyma, a surgical approach, she recommended at the Hawaii Dermatology Seminar provided by Global Academy for Medical Education/Skin Disease Education Foundation.
For the background erythema of rosacea, which she described as “that pink face that’s always there,” the two alpha-adrenergic receptor agonists available, brimonidine gel 0.33%, approved by the Food and Drug Administration in 2013, and oxymetazoline cream 1%, approved in 2017, both work on the neurovascular junction, but on different receptors.
Brimonidine “kicks in very, very rapidly,” with a significant decrease in background erythema evident within 30 minutes and improvements that last over a 12-hour day, she said. It is effective over a year, but in longterm and postmarketing studies, about 20% of patients experienced exacerbation of erythema, with two peaks of redness. “One occurs at 3-6 hours,” and the other peak occurs when the drug is wearing off later in the day, Dr. Stein Gold said.
A study that sought to identify factors that might make patients more prone to this adverse effect found that “less is better” regarding brimonidine application, with an optimal application of one to three pea-sized dollops on the face, not five as instructed in the package insert. In addition, patients with more than five flushing episodes a week, particularly women, “tend to have more labile disease and [are] more likely to get that rebound erythema,” the study found.
Oxymetazoline 1% in a cream formulation has a “slightly more gentle onset of action and a more gentle offset of action,” without exacerbation of erythema and has been shown to have sustained efficacy over 52 weeks. In a yearlong safety study, there were “no new red flags and we weren’t seeing that redness at hours 3 to 6, or even when you take the patient off the drug,” she noted.
Dr. Stein Gold reported that she has served as a consultant, investigator, or speaker for Galderma, Dermira, Foamix Pharmaceuticals, Valeant, Allergan, Actavis, and Roche.
SDEF/Global Academy for Medical Education and this news organization are owned by the same parent company.
LAHAINA, HAWAII – The pathophysiology of rosacea is complicated, which is why “we try to target our treatments to various areas in this pathogenic pathway” to achieve optimal results, according to Linda Stein Gold, MD, director of dermatology research at the Henry Ford Health System in Detroit.
For example, in a patient with papules and pustules, a topical or oral anti-inflammatory agent is needed “to calm that down.” If background erythema is present, separate from papules and pustules, use a topical alpha-adrenergic agonist, she advised. For telangiectasias, consider a device-based treatment, and for a phyma, a surgical approach, she recommended at the Hawaii Dermatology Seminar provided by Global Academy for Medical Education/Skin Disease Education Foundation.
For the background erythema of rosacea, which she described as “that pink face that’s always there,” the two alpha-adrenergic receptor agonists available, brimonidine gel 0.33%, approved by the Food and Drug Administration in 2013, and oxymetazoline cream 1%, approved in 2017, both work on the neurovascular junction, but on different receptors.
Brimonidine “kicks in very, very rapidly,” with a significant decrease in background erythema evident within 30 minutes and improvements that last over a 12-hour day, she said. It is effective over a year, but in longterm and postmarketing studies, about 20% of patients experienced exacerbation of erythema, with two peaks of redness. “One occurs at 3-6 hours,” and the other peak occurs when the drug is wearing off later in the day, Dr. Stein Gold said.
A study that sought to identify factors that might make patients more prone to this adverse effect found that “less is better” regarding brimonidine application, with an optimal application of one to three pea-sized dollops on the face, not five as instructed in the package insert. In addition, patients with more than five flushing episodes a week, particularly women, “tend to have more labile disease and [are] more likely to get that rebound erythema,” the study found.
Oxymetazoline 1% in a cream formulation has a “slightly more gentle onset of action and a more gentle offset of action,” without exacerbation of erythema and has been shown to have sustained efficacy over 52 weeks. In a yearlong safety study, there were “no new red flags and we weren’t seeing that redness at hours 3 to 6, or even when you take the patient off the drug,” she noted.
Dr. Stein Gold reported that she has served as a consultant, investigator, or speaker for Galderma, Dermira, Foamix Pharmaceuticals, Valeant, Allergan, Actavis, and Roche.
SDEF/Global Academy for Medical Education and this news organization are owned by the same parent company.
LAHAINA, HAWAII – The pathophysiology of rosacea is complicated, which is why “we try to target our treatments to various areas in this pathogenic pathway” to achieve optimal results, according to Linda Stein Gold, MD, director of dermatology research at the Henry Ford Health System in Detroit.
For example, in a patient with papules and pustules, a topical or oral anti-inflammatory agent is needed “to calm that down.” If background erythema is present, separate from papules and pustules, use a topical alpha-adrenergic agonist, she advised. For telangiectasias, consider a device-based treatment, and for a phyma, a surgical approach, she recommended at the Hawaii Dermatology Seminar provided by Global Academy for Medical Education/Skin Disease Education Foundation.
For the background erythema of rosacea, which she described as “that pink face that’s always there,” the two alpha-adrenergic receptor agonists available, brimonidine gel 0.33%, approved by the Food and Drug Administration in 2013, and oxymetazoline cream 1%, approved in 2017, both work on the neurovascular junction, but on different receptors.
Brimonidine “kicks in very, very rapidly,” with a significant decrease in background erythema evident within 30 minutes and improvements that last over a 12-hour day, she said. It is effective over a year, but in longterm and postmarketing studies, about 20% of patients experienced exacerbation of erythema, with two peaks of redness. “One occurs at 3-6 hours,” and the other peak occurs when the drug is wearing off later in the day, Dr. Stein Gold said.
A study that sought to identify factors that might make patients more prone to this adverse effect found that “less is better” regarding brimonidine application, with an optimal application of one to three pea-sized dollops on the face, not five as instructed in the package insert. In addition, patients with more than five flushing episodes a week, particularly women, “tend to have more labile disease and [are] more likely to get that rebound erythema,” the study found.
Oxymetazoline 1% in a cream formulation has a “slightly more gentle onset of action and a more gentle offset of action,” without exacerbation of erythema and has been shown to have sustained efficacy over 52 weeks. In a yearlong safety study, there were “no new red flags and we weren’t seeing that redness at hours 3 to 6, or even when you take the patient off the drug,” she noted.
Dr. Stein Gold reported that she has served as a consultant, investigator, or speaker for Galderma, Dermira, Foamix Pharmaceuticals, Valeant, Allergan, Actavis, and Roche.
SDEF/Global Academy for Medical Education and this news organization are owned by the same parent company.
REPORTING FROM SDEF HAWAII DERMATOLOGY SEMINAR
Celebrating 50 years of Dermatology News
The first issue of Skin & Allergy News, now Dermatology News, was published in January 1970. One front-page story highlighted the "continued improvement and more widespread use of steroids" as the most important development of the 1960s in dermatology. Another covered the launch of a national program for dermatology "to design a pattern for its future instead of simply drifting and letting its fate be determined by others."
Throughout 2020, look for articles and features marking the publication's golden anniversary. And read the first ever issue in the PDF above.
The first issue of Skin & Allergy News, now Dermatology News, was published in January 1970. One front-page story highlighted the "continued improvement and more widespread use of steroids" as the most important development of the 1960s in dermatology. Another covered the launch of a national program for dermatology "to design a pattern for its future instead of simply drifting and letting its fate be determined by others."
Throughout 2020, look for articles and features marking the publication's golden anniversary. And read the first ever issue in the PDF above.
The first issue of Skin & Allergy News, now Dermatology News, was published in January 1970. One front-page story highlighted the "continued improvement and more widespread use of steroids" as the most important development of the 1960s in dermatology. Another covered the launch of a national program for dermatology "to design a pattern for its future instead of simply drifting and letting its fate be determined by others."
Throughout 2020, look for articles and features marking the publication's golden anniversary. And read the first ever issue in the PDF above.