The Food and Drug Administration (FDA) has accepted for review the Biologic License Application (BLA) for fidanacogene elaparvovec for the treatment of adults with hemophilia B.
Fidanacogene elaparvovec is an investigational one-time gene therapy that contains a bio-engineered adeno-associated virus (AAV) capsid (protein shell) and a high-activity variant of human coagulation Factor IX (FIX) gene.
The application is supported by data from the open-label, single-arm phase 3 BENEGENE-2 study (ClinicalTrials.gov Identifier: NCT03861273), which evaluated the efficacy and safety of fidanacogene elaparvovec in adult males 18 to 65 years of age with moderately severe to severe hemophilia B (defined as FIX circulating activity of 2% or less).
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Eligible patients were required to have completed 6 months of routine FIX prophylaxis therapy during the lead-in study (ClinicalTrials.gov Identifier: NCT03587116) and then received a single intravenous (IV) infusion of fidanacogene elaparvovec. The primary endpoint was the annualized bleeding rate (ABR) for total bleeds after fidanacogene elaparvovec infusion vs FIX prophylaxis, administered as part of usual care.
Results showed after a single dose of fidanacogene elaparvovec, there was a 71% reduction in ABR, from an ABR of 4.43 during the lead-in pretreatment period to 1.3 from week 12 to month 15 (P <.0001). Fidanacogene elaparvovec was found to be generally well tolerated.
“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, MD, PhD, Chief Development Officer, Oncology and Rare Disease, Pfizer Global Product Development. “We look forward to continuing to work with global regulatory authorities to bring this innovative potential treatment to patients as quickly as possible.”
A Prescription Drug User Fee Act goal date has been set for the second quarter of 2024.
This article originally appeared on MPR
