FDA Green Lights Pfizer's Gene Therapy For Rare Bleeding Disorder Hemophilia

Friday, the FDA approved Pfizer Inc’s PFE Beqvez (fidanacogene elaparvovec-dzkt) for moderate to severe hemophilia B in adult patients who currently use factor IX (FIX) prophylaxis therapy or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var capsid. 

Beqvez is a one-time treatment designed to enable hemophilia B patients to produce FIX themselves rather than the current standard of care, which requires regular intravenous infusions of FIX that are often administered multiple times a week or multiple times a month.

Hemophilia B is a rare genetic bleeding disorder that prevents normal blood clotting because of a deficiency in FIX that causes those with the disease to bleed more frequently and longer than others.

The approval is based on the BENEGENE-2 Phase 3A study, which showed that a single dose of Pfizer’s therapy helped eliminate bleeding in 60% of patients​​ compared to 29% in the prophylaxis arm.

A median annualized bleeding rate (ABR) of zero (range of 0 to 19) was observed during the efficacy evaluation period compared to the prophylaxis arm, in which a median ABR of 1.3 (range of 0 to 53.9) was observed.

Pfizer said it will continue to monitor the long-term durability and safety of the treatment over 15 years.

With Beqvez now approved for use, Pfizer is launching a warranty program based on the durability of patient response to treatment. 

The warranty aims to provide greater certainty to payers, maximize access for eligible patients who receive Beqvez, and offer financial protection by insuring against the risk of efficacy failure.

Beqvez is currently under review with the European Medicines Agency, and the treatment recently received regulatory approval in Canada. 

Reuters noted that Pfizer has set a list price for Beqvez at $3.5 million in the U.S., similar to Australian drugmaker CSL Ltd’s CSLLY rival gene therapy Hemgenix.

Price Action: PFE shares are up 0.88% at $25.48 at the last check Friday.

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