NEW YORK – Pfizer's gene therapy for hemophilia B, fidanacogene elaparvovec, is under regulatory review in the US and EU, the company said Tuesday.
The US Food and Drug Administration has accepted Pfizer's biologics license application for the investigational therapy, while the European Medicines Agency has accepted Pfizer's marketing authorization application for the therapy, designed to treat adults with the rare genetic bleeding disorder.
Fidanacogene elaparvovec is an adeno-associated virus vector-based gene therapy that contains a variant of the FIX gene, which produces the clotting protein FIX that is deficient in people with hemophilia B. The treatment is designed to enable patients to produce the protein themselves, without needing intravenous infusions of FIX, which is currently the standard care.
Pfizer's regulatory submissions were based on findings from its Phase III BENEGENE-2 trial, in which 45 adult male patients with moderately severe to severe hemophilia B had completed at least six months of FIX prophylaxis therapy during a previous lead-in Phase III study.
Participants received one intravenous dose of the gene therapy. Based on a 15-month data cutoff, the therapy met its primary endpoint of non-inferiority and superiority in annualized bleeding rate after administration compared to intravenous infusions of FIX. Participants will continue to be followed for up to 15 years.
New York-based Pfizer in 2014 licensed fidanacogene elaparvovec from Philadelphia-based gene therapy developer Spark Therapeutics, which has since been acquired by Roche. As part of the licensing agreement, Pfizer is responsible for pivotal studies, regulatory activities, and global commercialization of the gene therapy.
The FDA is aiming to decide whether to grant approval to fidanacogene elaparvovec in Q2 of next year, according to Pfizer.