Pfizer heralds “paradigm shift” in hemophilia B gene therapy with FDA approval

By Jonathan Smith

- Last updated on GMT


Related tags Pfizer Hemophilia Spark Therapeutics Gene therapy adeno-associated virus

Pfizer's groundbreaking $3.5 million gene therapy for hemophilia B just got FDA approval. Here’s what it means for patients and the future of medicine

Key decision makers will need time to get familiar with working with groundbreaking gene therapies in hemophilia B care, Pfizer says, as the US Food and Drug Administration (FDA) has greenlit its gene therapy for the rare condition.

The approval of Beqvez (fidanacogene elaparvovec-dzkt), which is indicated for adults with moderate to severe forms of hemophilia B who currently use prophylaxis therapy or have life-threatening bleeding, is the second of its kind, with CSL Behring and uniQure’s Hemgenix authorized by the FDA in late 2022. Pfizer has set the price of Beqvez to a huge $3.5 million, the same as Hemgenix.

The standard approach to preventing bleeds in hemophilia B is regular infusions of the blood clotting Factor IX (FIX), which is impaired by a mutation in people with the rare condition. Beqvez is designed to reduce the need for regular FIX infusions by delivering a copy of the healthy FIX gene into patients via an adeno-associated virus (AAV) vector, and is indicated only for patients lacking antibodies against the viral vector.

“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,” said Adam Cuker, Director of Penn Comprehensive and Hemophilia Thrombosis Program, in Pfizer’s press release. “A one-time treatment with Beqvez has the potential to be transformative for appropriate patients by reducing both the medical and treatment burden over the long term.”

Commercial rollout of Beqvez

As Beqvez enters the US market, Pfizer plans to produce the therapy at a plant in Sanford, North Carolina, a company representative told this publication, adding that there is “a clear interest and anticipation from the physician and patient community” around the therapy.

“Given the ground-breaking nature of this paradigm shift in hemophilia B care, the decision to receive a gene therapy requires familiarity that we expect will grow over time and a shared decision-making approach among the key decision makers involved, particularly patients and their doctors,” said the company spokesperson.

Nonetheless, the first gene therapies for hemophilia face obstacles to commercialization in the US as the logistics and high prices put off many medical systems and health insurers from embracing the products.

In an attempt to navigate the issue, Pfizer is launching a warranty program linked to how long the treatment benefits patients to provide more certainty to payers and boost patient access to the therapy.

“We have taken an innovative approach that will continue to provide coverage if a patient changes insurance after they receive treatment for commercially insured patients,” said the company representative. “This was informed by pre-approval information exchange discussions we have had with payers to understand how we can best ensure access.”

Pfizer snapped up Beqvez when it took over Spark Therapeutics in 2014. With the commercial rollout underway in the US, the big pharma company is also pushing for market approval of the gene therapy in the EU and has candidates for other rare diseases in phase 3 such as hemophilia A and Duchenne muscular dystrophy.

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