FDA approves Roctavian for treatment of adults with severe hemophilia A
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FDA approved valoctocogene roxaparvovec-rvox for the treatment of certain adults with severe hemophilia A, according to the agent’s manufacturer.
The approval applies to individuals without antibodies to adeno-associated virus serotype 5 detected through an FDA-approved test.
Valoctocogene roxaparvovec-rvox (Roctavian, BioMarin Pharmaceutical) is an adeno-associated virus vector-based gene therapy administered as a one-time infusion. It delivers a functional gene designed to enable the body to produce factor VIII on its own.
Valoctocogene roxaparvovec-rvox is the first gene therapy to receive approval in the U.S. for patients with severe hemophilia A, according to a BioMarin press release.
"Adults with severe hemophilia A face a lifelong burden, with frequent infusions and a high risk [for] health complications, including uncontrolled bleeding and irreversible joint damage," Steven Pipe, MD, professor of pediatrics and pathology at University of Michigan and an investigator in the phase 3 study, said in a press release. “The approval of Roctavian as the first gene therapy for severe hemophilia A has the potential to transform the way we treat adults based on years of bleed control following a single, one-time infusion.”
The FDA approved the agent based on data from the phase 3 GENEr8-1 study.
As Healio previously reported, the open-label, single-group, multicenter trial included 134 men with severe hemophilia A who received the gene therapy at a dose of 6 x 10 13 vector genomes/kg and were followed for at least 3 years. Among them, 112 patients had baseline annualized bleeding rate data prospectively collected while on congenital factor VIII prophylaxis for at least 6 months. Those patients demonstrated a 52% reduction in mean annualized bleeding rate after receiving valoctocogene roxaparvovec-rvox through end of follow-up — 2.6 bleeds per year compared with 5.4 bleeds per year among those who received routine congenital factor VIII prophylaxis.
Researchers also reported a reduction in the rate of spontaneous bleeds and joint bleeds following treatment with valoctocogene roxaparvovec-rvox, with observed mean annualized bleeding rates of 0.5 spontaneous bleeds and 0.6 joint bleeds per year compared with baseline rates of 2.3 spontaneous bleeds and 3.1 joint bleeds per year while receiving congenital factor VIII prophylaxis.
BioMarin will conduct an extension study to monitor the long-term effects of therapy for up to 15 years, in addition to postapproval studies to track patients in a real-world setting for at least 15 years, according to the press release.
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