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Pfizer hemophilia gene therapy arrives in US to uncertain future

Dive Brief:

  • The Food and Drug Administration on Friday approved a new gene therapy for hemophilia, clearing Pfizer’s Beqvez for certain people with the less common “B” form of the bleeding condition.
  • Beqvez is for adults with moderate to severe hemophilia B who currently use drugs to prevent bleeds or have repeated, spontaneous bleeding. Eligible individuals also must be tested to determine whether they have antibodies that neutralize Beqvez’s effects.
  • Pfizer set the treatment’s list price at $3.5 million, a company spokesperson confirmed. That matches the cost of Hemgenix, the other available gene therapy for hemophilia B. Pfizer will offer insurers a warranty providing “financial protections” if Beqvez doesn’t work or its effects don’t last, the spokesperson wrote in an email, without providing details.

Dive Insight:

The FDA approval of Beqvez was years in the making for Pfizer, which has sought to have a presence in gene therapy.

The treatment came to Pfizer through a licensing agreement with Spark Therapeutics — now a unit of Roche — a decade ago. It was one of several deals Pfizer struck to build a gene therapy portfolio, and is the first to yield a marketed product, with approvals in Canada and now the U.S. The company also has experimental therapies for Duchenne muscular dystrophy and hemophilia A in late-stage testing, although it sold off other earlier-stage projects to AstraZeneca.

“The road to this breakthrough has been a 40+ year effort in which we have worked to advance the hemophilia treatment paradigm,” wrote Mikael Dolsten, Pfizer’s top scientist and head of research and development, in a post on LinkedIn.

Beqvez arrives as existing hemophilia gene therapies have fallen short of expectations.

Last year, the FDA approved two other, similar treatments: CSL and UniQure’s Hemgenix, and BioMarin Pharmaceutical’s Roctavian for hemophilia A. Both were seen as would-be blockbuster medicines, showing in testing the ability to keep bleeding in check for years. They were also important test cases for the economic viability of gene therapies — pitched as long-lasting alternatives to existing treatments and financial bargains for the healthcare system despite their high prices.

Yet, at least so far, their adoption has been slow. Sales of Hemgenix haven’t been large enough for CSL to break out in investor presentations. Roctavian only generated $3.5 million in sales in 2023 and $800,000 in the first three months of 2024, causing BioMarin to weigh divesting the drug altogether. CEO Alexander Hardy cited the “complexity” of selling Roctavian, a multi-step process that requires a “motivated patient” as well as supportive insurers and physicians.

“The handwriting is on the wall regarding Roctavian,” Piper Sandler analyst Christopher Raymond wrote in a note earlier this week, calling the drug a “commercial flop.”

Developers have urged patience. On an earnings call in February, CSL executives claimed patient referrals have been accelerating and said the company is confident more people will receive its drug as a result. BioMarin hasn’t given up entirely either. On a Wednesday call with investors, Hardy said the company remains focused on understanding “what the Roctavian opportunity is.”

Aamir Malik, Pfizer’s chief U.S. commercial officer, said in a Friday statement that Pfizer will lean on its “more than 40 years of experience in the hemophilia space.”

The company is “proactively working with treatment centers, payers, and the hemophilia community to appropriately help ensure the healthcare system is prepared to readily deliver Beqvez to the patients who can benefit from it,” Malik said.

Beqvez is currently under review in Europe. Pfizer also has an antibody drug for hemophilia A and B that’s being evaluated by regulators in the U.S. and Europe.