US FDA Approved First Gene Therapy to Treat Hemophilia B


CSL, a global biotechnology leader announced that the US Food and Drug Administration (FDA) approved HEMGENIX, the first one-time gene therapy to treat patients with hemophilia B. HEMGENIX is used to treat adults with hemophilia B who currently use factor IX prophylaxis therapy or have life-threatening haemorrhages.

In the clinical trial, HEMGENIX therapy decreased the rate of annual bleeding, and 94 percent of patients have stopped treatment with factor IX prophylaxis.

As part of the promise made to patients, CSL is committed to deliver innovative solutions to address several unmet medical needs, and CSL is proud to launch the next wave of innovative medicines for adults with hemophilia B, said Paul Perreault, MD and CEO of CSL.

Paul Perreault added that CSL recognize and is grateful to all clinical trial participants, investigators, and scientists, without whom this achievement would not be possible, and they are looking forward to seeing positive impact of the therapy on the hemophilia B patients.

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Kim Phelan, COO of The Coalition for Hemophilia B said they are very happy to experience this achievement in treating hemophilia B. Kim Phelan added that over the years they have seen different advancements for the hemophilia B community, but this gene therapy is the first ever treatment that offer the possibility of freedom from the need for ongoing and regular infusions of the hemophilia B patients.

Hemophilia B is a rare and life-threatening bleeding disorder caused by a single gene defect that result in insufficient production of a protein known as factor IX, which is produced by the liver that helps in forming blood clots.