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Pfizer On Wednesday, the company announced an experimental gene therapy. Rare inherited blood clotting disorders Successful Large-scale late-stage trialsPaving the way for possible approval.
The hemophilia A treatment could be the company’s second gene therapy drug to hit the U.S. market, following Bekbez. clear In April, I was tested for a less common type of bleeding disorder called hemophilia B.
Pfizer is promoting this treatment Sangamo TherapeuticsThe company’s shares rose more than 60% on Wednesday following the data release, while Pfizer shares were flat on Wednesday.
Pfizer is one of several pharmaceutical companies investing in the fast-growing field of gene and cell therapies, expensive, one-time treatments that target a patient’s genetic material or cells to cure or significantly alter the course of a disease. Some industry medical experts predict such treatments could replace the lifelong treatments patients have traditionally received to manage chronic conditions.
Hemophilia A is a lifelong disease caused by a deficiency of proteins called blood clotting factors. Without enough of these proteins, blood cannot clot properly, increasing the risk of spontaneous bleeding or severe bleeding after surgery. Based on data, Pfizer said the disease occurs in approximately 25 of every 100,000 male births worldwide.
Pfizer announced that after 15 months, the one-time treatment significantly reduced the number of annual bleeding episodes in people with moderate to severe hemophilia A. The company also said the drug worked better than the current standard of care for the disease, which is regular infusions to replace the factor VIII protein.
“For people with hemophilia A, the physical and psychological impact of needing frequent infusions and injections to prevent and treat bleeding cannot be underestimated,” Dr. Andrew Leavitt, the trial’s lead investigator, said in a statement.
Pfizer said the study is continuing and it plans to present additional data at an upcoming medical conference.
If approved, Pfizer’s treatment would Biomarine PharmaceuticalsBioMarin’s treatment has been rolled out slowly since it was approved in the United States last year, raising questions about how many patients will take Pfizer’s drug if it hits the market.
Biomarine is In consideration of Whether to sell a hemophilia A drug that costs $2.9 million.
