The U.S. Food and Drug Administration (FDA) has pushed back by more than one year a decision on Roctavian, the potential first gene therapy for adults with hemophilia A. In a complete response letter that took the therapy’s developer, BioMarin by surprise, the FDA said it needed…
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The European Medicines Agency (EMA) has accepted BioMarin Pharmaceutical’s request for accelerated assessment of Roctavian, the company’s investigational gene therapy for severe hemophilia A, for a second time. Last year, BioMarin’s regulatory applications for Roctavian (valoctocogene roxaparvovec) got rejected both in Europe and in the…
A 39-year-old man from Arizona became the first patient in Nevada to receive Hemgenix (etranacogene dezaparvovec), the first gene therapy to be approved for hemophilia B. The infusion was administered at the Cure 4 The Kids Foundation, under the supervision of Aimee Foord, director of the foundation’s bleeding…
For physically active men with severe hemophilia A, switching from their standard preventive treatment to Hemlibra (emicizumab-kxwh) can help reduce bleeding, a recent study suggests. This change not only improved their quality of life but also reduced the burden of treatment, allowing them to maintain their active lives.
The biotech companies Swedish Orphan Biovitrum AB (Sobi) and Biogen have announced that the European Commission (EC) has approved their extended half-life therapy Alprolix (rFIXFc) for hemophilia B in all 28 European Union (EU) countries. In addition, the EC maintained the drug’s orphan designation. The World Federation of Hemophilia (WFH) estimated…
The gene therapy BBM-H901 (dalnacogene ponparvovec) has been approved in China for adults with moderate to severe hemophilia B, making it the first approved gene therapy for the disorder in the country. BBM-H901 was developed and will be manufactured by Belief Biomed (BBM), but it will be commercialized…
Two years of preventive treatment with Genentech’s Hemlibra (emicizumab) safely and effectively lowered the number of bleeds in people with hemophilia A and clotting factor VIII (FVIII) inhibitors, according to final data from the Phase 3b STASEY trial. Development of antibodies against the therapy (anti-drug antibodies) was…
Eloctate (efmoroctocog alfa) is an extended half-life therapy that's approved to prevent and treat bleeds, including those occurring during surgery, in adults and children with hemophilia A.
Albumin fusion is a molecular technology that has been used to create replacement therapies for hemophilia with an extended half-life — treatments that are more long-lasting in the body and can therefore be administered less frequently.
Note: This story was updated Feb. 24, 2023, to correct the secondary headline since there are several other recombinant factor VIII therapies available. Altuviiio is a first-in-class therapy, but not the first. Efanesoctocog alfa, Sobi and Sanofi’s first-in-class, long-lasting recombinant or man-made factor…