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Pfizer announced Wednesday that its experimental gene therapy for a rare genetic blood-clotting disorder was successful in a large late-stage trial, paving the way for possible approval.
The drug to treat hemophilia A could be the company's second gene therapy on the U.S. market, following Beqvez, which was approved in April to treat a less common type of blood clotting disorder called hemophilia B.
Pfizer is developing the therapy together with Sangamo Therapeuticswhose shares rose more than 60% on Wednesday after the data was released before giving back some of those gains. Pfizer's stock was essentially unchanged on Wednesday.
Pfizer is one of several pharmaceutical companies investing in the fast-growing field of gene and cell therapies – one-time, expensive treatments that target a patient's genetic source or cell to cure a disease or significantly alter its course. Some industry experts expect these therapies to replace the traditional lifelong treatments that patients take to manage chronic diseases.
Hemophilia A is a lifelong disease caused by a deficiency of the blood-clotting protein Factor VIII. Without sufficient amounts of this protein, blood cannot clot properly, increasing the risk of spontaneous bleeding and severe bleeding after surgery. The disease occurs in about 25 out of every 100,000 male births worldwide, Pfizer said in a press release, citing data.
Pfizer said its one-time treatment significantly reduced the number of annual bleeding episodes in patients with moderate to severe hemophilia A after 15 months. The company said the drug also worked better than the current standard treatment for the disease, which is routine infusions that replace the factor VIII protein.
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“For people with hemophilia A, the physical and emotional impact of the need to prevent and treat bleeding episodes through frequent intravenous infusions or injections cannot be underestimated,” Dr. Andrew Leavitt, the study's principal investigator, said in a statement.
Pfizer said the study was ongoing and that further data would be presented at future medical meetings.
If approved, Pfizer’s therapy will compete with BioMarin PharmaceuticalBioMarin's one-time treatment with Roctavian has had a slow start since it was approved in the U.S. last year, raising questions about how many patients would take Pfizer's drug when it comes to market.
BioMarin is reportedly considering divesting its $2.9 million hemophilia A therapy.
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