FDA Approves Pfizer’s Gene Therapy Beqvez for Hemophilia

The FDA has approved Pfizer’s one-time gene therapy Beqvez (fidanacogene elaparvovec-dzkt) to treat adults with moderate-to-severe hemophilia B. It is indicated for those who currently use factor IX (FIX) prophylaxis therapy, have current hemorrhage, repeated bleeding episode or do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid.

Hemophilia is a rare genetic bleeding disorder caused by a deficiency in one of several blood clotting factors. People with hemophilia B have a deficiency in clotting human coagulation Factor IX, a specific protein in the blood. The current standard of care requires recurrent intravenous infusions of either plasma-derived or recombinant Factor IX. More than 38,000 people worldwide are living with hemophilia B, according to the World Federation of Hemophilia.

“Many people with hemophilia B struggle with the commitment and lifestyle disruption of regular FIX infusions, as well as spontaneous bleeding episodes, which can lead to painful joint damage and mobility issues,” Adam Cuker, M.D., M.S., director, Penn Comprehensive and Hemophilia Thrombosis Program, said in a news release.

Beqvez is a one-time gene therapy that contains a bio-engineered adeno-associated virus (AAV) capsid and a high-activity variant of the Factor IX gene. Pfizer licensed the gene therapy from Spark Therapeutics. Bequez will be priced at $3.5 million, which is in line with the previously approved gene therapy for hemophilia B Hemgenix.

A Pfizer spokesperson said the price is based on a holistic view of its value, including the benefits generated for patients, healthcare systems, and society. Annual costs per individual may be more than $600,000 and as high as $1.1 million, not including indirect costs such as outpatient care and managing uncontrolled bleeds, the spokesperson said.

Adam Cuker, M.D.

“Beqvez is designed to provide years of consistent bleed control with a one-time dose, reducing the burden of frequent infusions. We are confident that Beqvez will provide long-term clinical benefits for people living with hemophilia B, as well as value to healthcare systems by reducing healthcare spend over time,” the spokesperson said.

Pfizer executives said they are working with treatment centers, payers, and the hemophilia community to ensure access. Pfizer is launching a warranty program based on durability of patient response to treatment with the goal of offering financial protection by insuring against the risk of efficacy failure, the news release said.

The spokesperson said Pfizer will continue to provide coverage if a patient changes insurance after they receive treatment for commercially insured patients, and the company anticipates a refund will be provided to the insurers. “This was informed by pre-approval information exchange discussions we have had with payers to understand how we can best ensure access.

“The program speaks to the confidence that we have in both our medicine’s ability to deliver for patients as well as value that it can offer the healthcare system. The offering is based on durability of patient response to treatment, and we anticipate it will offer financial protection for eligible patients by insuring against the risk of efficacy failure.”

For patients, Pfizer offers personalized patient support services including financial assistance resources and logistical support through the Pfizer Gene Together program, which provides support for several products including Beqvez. The maximum benefit for these programs ranges from $10,000 to $25,000 for out-of-pocket expenses including co-pays or coinsurances.

Although the terms of the assistance program don’t specifically mention accumulator or maximizer programs, it states that patients should not use the copay program if their insurer or health plan prohibits use of manufacturer co-pay assistance programs.

Related: FDA Sets Action Date for Hemophilia B Gene Therapy

The approval was based on the results from the pivotal BENEGENE-2 study, a phase 3, single-arm study that enrolled 45 adult men to evaluate the efficacy and safety of Beqvez. Patients will be followed for 15 years, including the six years in the study and an additional nine years in separate study to assess long-term safety and efficacy.

The BENEGENE-2 trial met its primary endpoint of non-inferiority in the annualize bleeding rate (ABR) of total bleeds post-infusion compared with prophylaxis regimen with factor IX. The most common adverse event for patients treated with Beqvez was an increase in transaminases, a group of enzymes that can impact liver function. No deaths, serious adverse events related to treatment or associated with infusion reactions, thrombotic events, or FIX inhibitors were reported.

Beqvez was approved in Canada earlier this year.

This is the second one-time gene therapy approved for hemophilia B. In November 2022, the FDA approved CSL Behring’s Hemgenix (etranacogene dezaparvovec). Hemgenix is also a adeno-associated virus vector-based gene therapy and has a price of $3.5 million.