The U.S. Food and Drug Administration (FDA) has accepted a resubmitted biologics license application (BLA) from BioMarin Pharmaceutical requesting approval of the gene therapy Roctavian (valoctocogene roxaparvovec) to treat adults with severe hemophilia A. The FDA is now expected to decide by the end of March 2023 on whether…

The U.S. Food and Drug Administration (FDA) has cleared the biotechnology company TeraImmune to launch a Phase 1/2a clinical trial testing TI-168, its regulatory T-cell therapy designed to eliminate inhibitors in people with hemophilia A. The upcoming trial is expected to enroll up to 18 people with congenital (genetic)…

Men with moderate-to-severe hemophilia A are again being enrolled in the Phase 3 AFFINE trial, which is testing the safety and effectiveness of SB-525 (giroctocogene fitelparvovec), an experimental gene therapy being developed by Pfizer and Sangamo Therapeutics. Pfizer, the study’s sponsor, had placed a voluntary pause…

If approved for hemophilia A, the one-time gene therapy Roctavian (valoctocogene roxaparvovec) would provide substantial cost savings per patient compared with other preventive treatments, specifically Hemlibra. That’s according to a draft evidence report issued by pricing watchdog the Institute for Clinical and Economic Review (ICER). An independent non-profit…

A participant in a Phase 3 clinical trial evaluating Roctavian (valoctocogene roxaparvovec), a gene therapy for hemophilia A, has been diagnosed with leukemia. A genetic assessment of the case, conducted by the therapy’s developer, BioMarin Pharmaceutical, suggested the cancer diagnosis was not linked to Roctavian. Details of…

The European Commission has granted conditional marketing authorization to BioMarin’s one-time gene therapy Roctavian (valoctocogene roxaparvovec) as a treatment for adults with severe hemophilia A who have neither inhibitors nor detectable antibodies against adeno-associated virus serotype 5 (AAV5). The decision, which comes a couple of months after a…

A single dose of Freeline Therapeutics’ experimental gene therapy FLT180a increased the levels of factor IX (FIX) — the missing clotting protein in hemophilia B — for up to around 3.5 years in nine out of 10 men with moderate-to-severe disease. That’s according to data from the Phase…

The first three participants in the B-LIEVE trial all have factor IX (FIX) levels within the normal range more than one month after being treated with FLT180a, Freeline’s experimental gene therapy for hemophilia B, according to the company. “The initial data show that FLT180a provides rapid and consistent…

Nearly one-third of people with hemophilia B have antibodies against a subtype of an adeno-associated virus (AAV) — called AAV6 — that is used as an experimental gene therapy carrier, a U.K. study found. Because such antibodies could render the treatment that the carrier holds ineffective, these patients would…

European authorities have recommended conditional marketing authorization for Roctavian (valoctocogene roxaparvovec), BioMarin Pharmaceutical’s one-time gene therapy for adults with severe hemophilia A. This positive decision, from the Committee for Medicinal Products for Human Use (CHMP), makes Roctavian the first gene therapy for hemophilia A to be recommended for European…