FDA approves Pfizer’s first gene therapy to treat rare inherited bleeding disorder

The US Food and Drug Administration officially recognized it on Friday PfizerThe rare genetic disease treatment of bleeding disorders is the first gene therapy developed by the company to win a US license

The company Sabuj Alo will market the drug as Bekvez to treat moderate to severe hemophilia B in adults who meet certain conditions.

More than 7,000 people in the United States suffer from this debilitating condition, according to advocacy groups. The condition is caused by insufficient levels of a certain protein that helps blood clot to stop bleeding and stop wounds. Without this protein, called factor IX, people with hemophilia B bruise easily and bleed more frequently and last longer.

Bekvez is a one-time treatment designed to enable patients to make their own factor IX and prevent and control bleeding. In late-stage trials, the drug often outperformed the more difficult standard treatment for hemophilia B, the protein intravenously a few times a week or month. is given

“Many hemophilia B patients struggle with the commitment and lifestyle disruption of regular (factor IX) infusions, as well as spontaneous bleeding, which can lead to painful joint damage,” said Dr. Adam Kuker, director of the University of Pennsylvania General Hospital. and Action Matters” and the Hemophilia Thrombosis Program, Pfizer. Released Friday.

Cook added that Pfizer’s drug “has the potential to be transformative for the right patients by reducing long-term medical and treatment burdens.”

The approval is a big step for Pfizer, which is trying to regain its footing after a sharp decline in business last year due to the coronavirus pandemic. The company is betting on cancer drugs and treatments for other diseases to help turn around its business.

Pfizer is one of several companies investing in the fast-growing field of gene and cell therapy. These are one-time, high-cost treatments that aim to cure a patient’s genetic origins or cells or significantly alter the course of the disease. Some health experts hope that cell and gene therapies will replace the traditional lifelong treatments people use to manage chronic diseases.

In 2014, Pfizer acquired the rights to manufacture and market Spark Therapeutics BakeVege. The company did not reveal how the treatment will cost.

Gene therapy will compete with Australia’s CSL Behring for a Hermogenics-like process to get FDA approval to treat hemophilia B in 2022. In the United States, the drug costs up to $3.5 million before insurance and other discounts.

Notably, some health experts say factors such as high costs and logistical problems have led to limited uptake of HemGenics and another approved gene therapy for the more common form of hemophilia A.

Pfizer is also seeking FDA approval for its experimental antibody, marstacimab, to treat hemophilia A and B. The company is also developing a gene therapy for Duchenne muscular dystrophy, a genetic disorder that causes the muscles to gradually weaken.