A Gene Therapy Discontinued

Less than a year after FDA approval, Beqvez, a gene therapy for hemophilia B, has been discontinued. Manufactured by Pfizer, the one-time treatment carried a list price of $3.5 million per person. Several reasons led to the discontinuation, including limited interest from patients and doctors toward hemophilia gene therapies to date. Gene therapy for hemophilia has struggled to find a place in the market, due to its high price, and the current plethora of effective alternative treatments.

Source: LA Kelley Communications, Inc., Communique (March 2025)