Sanofi ALTUVIIIO hemophilia A treatment: FDA accepts sBLA based on pediatric phase 3 data

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The FDA has accepted Sanofi’s supplemental Biologics License Application (sBLA) for ALTUVIIIO, a novel, high-sustained factor VIII replacement therapy for adults and children with hemophilia A. The FDA based the decision on positive final data from the pivotal Phase 3 XTEND-Kids trial in children under 12 with hemophilia A.

ALTUVIIIO (efanesoctocog alfa) is the first Factor VIII replacement therapy to offer bleed protection with once-weekly dosing. In February, Sanofi announced that FDA had approved the drug for routine prophylaxis and on-demand treatment to control bleeding episodes. Its use also extends to perioperative management (surgery) for adults and children with hemophilia A.

Sanofi developed in collaboration with Sobi. Barclays projects that the drug will top €2 billion in peak sales.

Supportive findings from XTEND-kids study

The current FDA approval is supporte…

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  • September 12, 2023
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Roche shares upbeat Phase 3 data for Hemlibra in hemophilia

Facing potential competition from Novo Nordisk’s (NYSE:NVO) investigational blood coagulation factor stimulants Mim8, Roche (SIX:RO, ROG; OTCQX:RHHBY) released positive new data from the Phase 3 HAVEN 6 study focused on the use of Hemlibra (emicizumab) in patients with mild or moderate hemophilia A.

Hemlibra won FDA approval in 2018 for hemophilia A without factor VIII inhibitors.

The drug raked in almost $2.8 billion in sales in 2021.

In the open-label, single-arm HAVEN 6 study, two-thirds of Hemlibra recipients with mild or moderate hemophilia A had no treated bleeds at 55.6 weeks median follow-up.

Basel, Switzerland–based Roche said the drug continued to have a favorable safety profile in patients without factor VIII inhibitors.

The company plans on presenting the data at the International Society on Thrombosis and Haemostasis (ISTH) Annual Congress on July 11, 2022 in London.

Roche intends to ask the European Medicines Agency to …

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  • July 12, 2022
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