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Pfizer said on Wednesday that its experimental gene therapy Rare inherited blood clotting disorder successfully performed once Large late-stage trialpaving the way for potential approval.
The treatment for hemophilia A could become the company’s second gene therapy to enter the U.S. market, following Beqvez, which was approved in April to treat a less common bleeding disorder called hemophilia B. sick.
Pfizer is developing this therapy with: Sangamo therapyShares rose more than 60% on Wednesday after the data was released. Pfizer’s shares were flat Wednesday.
Pfizer is one of several drugmakers investing in the fast-growing field of gene and cell therapies, which are one-time, expensive treatments that target a patient’s genetic source or cells to cure or significantly change the course of a disease. Some industry health experts expect these therapies to replace the traditional lifelong therapies patients use to manage chronic conditions.
Hemophilia A is a lifelong disease caused by a lack of a blood clotting protein called factor VIII. Without enough protein, the blood cannot clot properly, increasing the risk of spontaneous bleeding and severe bleeding after surgery. Globally, about 25 out of every 100,000 male births are affected by the disease, Pfizer said in a news release, citing data.
Pfizer said its one-time treatment significantly reduced the number of annual bleeds in patients with moderate to severe hemophilia A after 15 months. The drug also works better than the current standard treatment for the disease, which replaces conventional infusions of the factor VIII protein, the company said.
“For people with hemophilia A, the need to prevent and treat bleeding events with frequent intravenous fluids or injections can have physical and emotional consequences,” Dr. Andrew Levitt, the trial’s principal investigator, said in a statement. Not to be underestimated.
Pfizer said the study is ongoing and more data will be presented at an upcoming medical meeting.
If approved, Pfizer’s treatment would be Biomarine PharmaceuticalsRoctavian’s one-time cure. BioMarin’s treatment has been slow to advance since it was approved in the U.S. last year, raising questions about how many patients would take Pfizer’s drug if it made it to the market.
According to reportsBioMarin considering Whether to divest its $2.9 million hemophilia A therapy.
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