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EMA's CHMP Issues Positive Opinion on Pfizer's Hemophilia B Gene Therapy

NEW YORK – The European Medicines Agency on Friday said its Committee for Medicinal Products for Human Use (CHMP) has recommended granting conditional marketing authorization in the EU to Pfizer's hemophilia B gene therapy Durveqtix (fidanacogene elaparvovec).

Durveqtix, which is branded as Beqvez in the US and Canada where it is already approved, is designed as a treatment for adults with severe and moderately severe hemophilia B and who do not have factor IX inhibitors nor detectable antibodies to variant adeno-associated virus serotype Rh74, which is the vector used in the treatment to deliver a functional copy of the FIX gene.

Hemophilia B is a rare and inherited bleeding disorder caused by a deficiency in coagulation factor IX (FIX), a clotting protein produced by the FIX gene.

As a one-time gene therapy, Durveqtix aims to enable sustained production of FIX.

After reviewing available data, the EMA's Committee for Advanced Therapies (CAT), an expert committee for cell- and gene-based medicines, also determined Durveqtix's benefits outweighed possible risks for patients with hemophilia B.

The recommendations from CHMP and CAT are based on results from an ongoing single-arm, open-label Phase III trial in which Durveqtix substantially reduced the frequency of bleeds compared to a lead-in period in which patients were treated with standard care, which involved a routine FIX prophylaxis regimen.

The annualized bleeding rate was 1.44 for patients treated with gene therapy compared to 4.5 when they were treated with the FIX prophylaxis regimen. Sixty percent of patients remained free of bleeding events through their individual follow-up period, which ranged from two to four years, and consumption of prophylactic FIX was reduced by 92 percent after gene therapy.

As part of the study, patients will be monitored for up to 15 years.

Common side effects included increased levels of liver enzymes, headache and flu-like symptoms, and increased levels of creatinine and lactate dehydrogenase.

The European Commission will next review CHMP's opinion on Durveqtix and decide whether to make these treatments available to patients in the European Union.