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The Food and Drug Administration has approved efanesoctocog alfa (Altuviiio), a first-in-class, high-sustained factor VIII replacement therapy for adults and children with hemophilia A.

The product is used once a week and is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as to control bleeding during surgery (perioperative management).

The manufacturer, Sanofi, says that this is “the first and only hemophilia A treatment that delivers normal to near-normal factor activity levels (over 40%) for most of the week with once-weekly dosing, and significantly reduces bleeds compared to prior factor VIII prophylaxis.”

With this product, “we have the opportunity to provide near-normal factor activity levels for an extended period of time (the majority of a week) with a single dose, which is a first for hemophilia A,” said Angela Weyand, MD, of Michigan Medicine, who was involved in the pivotal phase 3 XTEND-1 trial that led to approval.

Results from the XTEND-1 trial were recently published online in The New England Journal of Medicine. The results show that one injection of efanesoctocog alfa, a factor VIII therapy, resolved almost all bleeding episodes (97%) in the overall patient population and weekly prophylaxis provided mean factor VIII activity in the normal or near-normal range (> 40 IU/dL) for most of the week, as previously reported by this news organization.

The researchers noted that the factor VIII therapies that have been available up to now need to be administered frequently.

In an accompanying editorial, Cindy Leissinger, MD, called efanesoctocog alfa a “victory” for patients with hemophilia A.

“In a crowded field of transformative therapies for hemophilia, efanesoctocog alfa stands out as a winner – a major therapeutic advance that achieves highly protective factor VIII levels with a once-weekly infusion,” wrote Dr. Leissinger, director of the Louisiana Center for Bleeding and Clotting Disorders at Tulane University in New Orleans.

Sanofi said that Altuviiio is expected to be commercially available in April 2023. The company said that it will price the product at parity to the annual cost of treating a prophylaxis patient with its Eloctate product (antihemophilic factor [recombinant], Fc fusion protein). Sanofi will provide comprehensive patient support services and resources online and at 1-855-MyALTUVIIIO (855-692-5888).

A version of this article originally appeared on Medscape.com.

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The Food and Drug Administration has approved efanesoctocog alfa (Altuviiio), a first-in-class, high-sustained factor VIII replacement therapy for adults and children with hemophilia A.

The product is used once a week and is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as to control bleeding during surgery (perioperative management).

The manufacturer, Sanofi, says that this is “the first and only hemophilia A treatment that delivers normal to near-normal factor activity levels (over 40%) for most of the week with once-weekly dosing, and significantly reduces bleeds compared to prior factor VIII prophylaxis.”

With this product, “we have the opportunity to provide near-normal factor activity levels for an extended period of time (the majority of a week) with a single dose, which is a first for hemophilia A,” said Angela Weyand, MD, of Michigan Medicine, who was involved in the pivotal phase 3 XTEND-1 trial that led to approval.

Results from the XTEND-1 trial were recently published online in The New England Journal of Medicine. The results show that one injection of efanesoctocog alfa, a factor VIII therapy, resolved almost all bleeding episodes (97%) in the overall patient population and weekly prophylaxis provided mean factor VIII activity in the normal or near-normal range (> 40 IU/dL) for most of the week, as previously reported by this news organization.

The researchers noted that the factor VIII therapies that have been available up to now need to be administered frequently.

In an accompanying editorial, Cindy Leissinger, MD, called efanesoctocog alfa a “victory” for patients with hemophilia A.

“In a crowded field of transformative therapies for hemophilia, efanesoctocog alfa stands out as a winner – a major therapeutic advance that achieves highly protective factor VIII levels with a once-weekly infusion,” wrote Dr. Leissinger, director of the Louisiana Center for Bleeding and Clotting Disorders at Tulane University in New Orleans.

Sanofi said that Altuviiio is expected to be commercially available in April 2023. The company said that it will price the product at parity to the annual cost of treating a prophylaxis patient with its Eloctate product (antihemophilic factor [recombinant], Fc fusion protein). Sanofi will provide comprehensive patient support services and resources online and at 1-855-MyALTUVIIIO (855-692-5888).

A version of this article originally appeared on Medscape.com.

The Food and Drug Administration has approved efanesoctocog alfa (Altuviiio), a first-in-class, high-sustained factor VIII replacement therapy for adults and children with hemophilia A.

The product is used once a week and is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as to control bleeding during surgery (perioperative management).

The manufacturer, Sanofi, says that this is “the first and only hemophilia A treatment that delivers normal to near-normal factor activity levels (over 40%) for most of the week with once-weekly dosing, and significantly reduces bleeds compared to prior factor VIII prophylaxis.”

With this product, “we have the opportunity to provide near-normal factor activity levels for an extended period of time (the majority of a week) with a single dose, which is a first for hemophilia A,” said Angela Weyand, MD, of Michigan Medicine, who was involved in the pivotal phase 3 XTEND-1 trial that led to approval.

Results from the XTEND-1 trial were recently published online in The New England Journal of Medicine. The results show that one injection of efanesoctocog alfa, a factor VIII therapy, resolved almost all bleeding episodes (97%) in the overall patient population and weekly prophylaxis provided mean factor VIII activity in the normal or near-normal range (> 40 IU/dL) for most of the week, as previously reported by this news organization.

The researchers noted that the factor VIII therapies that have been available up to now need to be administered frequently.

In an accompanying editorial, Cindy Leissinger, MD, called efanesoctocog alfa a “victory” for patients with hemophilia A.

“In a crowded field of transformative therapies for hemophilia, efanesoctocog alfa stands out as a winner – a major therapeutic advance that achieves highly protective factor VIII levels with a once-weekly infusion,” wrote Dr. Leissinger, director of the Louisiana Center for Bleeding and Clotting Disorders at Tulane University in New Orleans.

Sanofi said that Altuviiio is expected to be commercially available in April 2023. The company said that it will price the product at parity to the annual cost of treating a prophylaxis patient with its Eloctate product (antihemophilic factor [recombinant], Fc fusion protein). Sanofi will provide comprehensive patient support services and resources online and at 1-855-MyALTUVIIIO (855-692-5888).

A version of this article originally appeared on Medscape.com.

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