FDA Accept Pfizer’s BLA for Hemophilia B Gene Therapy
28 June 2023
Regulatory & Standards
Pfizer has announced that the FDA has accepted its Biological License Application (BLA) for its gene therapy fidanacogene elaparvovec to treat adults with hemophilia B.
This investigational gene therapy, that has previously been granted Breakthrough, Regenerative Medicines Advanced Therapy (RMAT) and orphan drug designations from the FDA, is also currently under European marketing authorization application (MAA) review by the European Medicines Agency (EMA).
Built upon an AAV capsid, fidanacogene elaparvovec contains a high-activity variant of the human coagulation Factor IX (FIX) gene – the gene that is deficient in patients with hemophilia B.
The aim of this therapy is to enable recipients to self-produce FIX after just a one-time treatment, to replace the current standard of care for hemophilia B which involves regular intravenous infusions of FIX.
“Gene therapy marks a new era of scientific advancement, and if approved, we believe fidanacogene elaparvovec has the potential to transform the lives of people living with hemophilia B who are eligible for treatment,” said Chris Boshoff, Chief Development Officer, Oncology and Rare Disease, Pfizer Global Product Development.
“Patients are at the center of our legacy of innovation in hemophilia. Despite significant progress in their treatment, those living with hemophilia continue to experience disruption to daily life and need new options,” Chriss Boshoff adds.
The FDA and EMA submissions for fidanacogene elaparvovec are supported by 15-months of data from the open-label, single arm, Phase III BENEGENE-2 study.
45 adult males aged 18-65 with moderately severe to severe hemophilia B – defined as 2% or less of circulating FIX – enrolled in the study receiving a one-time intravenous dose of fidanacogene elaparvovec, following 6 months of routine FIX prophylaxis therapy.
The BENEGENE-2 study met its primary endpoint of non-inferiority and superiority in the annualized bleeding rate (ABR) of total bleeds post-fidanacogene elaparvovec infusion versus prophylaxis regimen with FIX.
It was also reported that the gene therapy was generally well-tolerated, with a safety profile consistent with Phase I/II results.
Participants in the trial are set to be followed for up to 15 years as Pfizer aims to learn more about the long-term impact of this gene therapy in patients with hemophilia B.
The FDA has set a goal date in the second quarter of 2024 for the Prescription Drug User Free Act (PDUFA) of fidanacogene elaparvovec.
“We look forward to continuing to work with global regulatory authorities to bring this innovative potential treatment to patients as quickly as possible,” concludes Chris.