Pfizer wins Canadian OK for hemophilia treatment, its first gene therapy

Pfizer wins Canadian OK for hemophilia treatment, its first gene therapy

  04 Jan 2024

Dive Brief:

  • Canada’s health regulators have approved Pfizer’s gene therapy for hemophilia B, the company said Wednesday, making it the first time the drugmaker has received regulatory clearance for such a genetic medicine.
  • The gene therapy, which Pfizer licensed nine years ago from Spark Therapeutics, will compete with CSL’s Hemgenix, a similar treatment that gained Canadian approval in October 2023. Called Beqvez, Pfizer’s therapy is also under review by the Food and Drug Administration, with a decision expected in the second quarter of 2024.
  • After investing heavily in gene therapy in the 2010s, Pfizer has backed away from the kind of treatment represented by Beqvez, which uses viral vectors to deliver a functional gene into the body. Its hemophilia A and Duchenne muscular dystrophy therapies have faced safety delays, and the drugmaker has missed out on becoming a first mover.

Dive Insight:

Hemophilia and other blood disorders like sickle cell disease and beta thalassemia have drawn intense investment from genetic medicine developers. in December, the FDA approved two such treatments for sickle cell, clearing Vertex Pharmaceuticals’ gene editing drug Casgevy and Bluebird bio’s viral vector therapy Lyfgenia.

In hemophilia B — the more uncommon of the two types of the bleeding disorder — Hemgenix has been on the U.S. market since November 2022, where it has a list price of $3.5 million. Australia-based CSL, which licensed the treatment from gene therapy pioneer UniQure, hasn’t broken out sales revenue from Hemgenix in its most recent financial reports.

In Canada, the two companies will be competing to treat around 700 people, some of whom may be successfully treated with factor protein replacements and might not seek out gene therapy.

Being on the market first is an advantage for drugmakers, and even more so for gene therapies intended to be one-time treatments. Early last year, when Pfizer announced its strategic shift, a company spokesperson said Pfizer wanted to “focus on programs where our innovation and investments are differentiated and where we are best positioned to generate high-impact medicines and vaccines.”

Those moves included focusing on genetic medicine tools other than viral vectors, putting a gene therapy facility up for sale, and in July selling off some programs and technology to AstraZeneca’s Alexion rare disease unit for up to $1 billion.

The bid to reduce its gene therapy spending came amid a tumultuous year for Pfizer, which announced plans to cut costs by around $4 billion in 2024, primarily from research and development, as plummeting sales of COVID-19 vaccines and drugs caused revenues to come in below the company’s expectations.

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