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On Friday, the Food and Drug Administration (FDA) revealed its approval of Pfizer’s treatment for a rare genetic bleeding disorder, marking the company’s debut gene therapy to receive clearance in the U.S. Marketed under the name Beqvez, the drug has been given the green light by the agency for adults with moderate to severe hemophilia B who fulfill specific criteria.
A Pfizer spokesperson informed CNBC that the treatment will be accessible to eligible patients via prescription in the current quarter. The spokesperson highlighted its substantial $3.5 million price tag before insurance and other rebates, positioning it as one of the most expensive drugs in the U.S. According to an advocacy group, over 7,000 individuals in the U.S. grapple with this debilitating condition, which predominantly affects males. The disorder stems from inadequate levels of a specific protein crucial for blood clotting to halt bleeding and seal wounds. Without this protein, known as factor IX, patients with hemophilia B experience heightened susceptibility to bruising and prolonged, frequent bleeding episodes.
Beqvez represents a one-time treatment engineered to stimulate patients’ production of factor IX, effectively preventing and managing bleeding episodes. In a late-stage trial, the drug demonstrated superiority over the often cumbersome standard treatment for hemophilia B, which necessitates frequent protein infusions administered via veins weekly or monthly. Adam Cuker, director of Penn Medicine’s Comprehensive and Hemophilia Thrombosis Program, expressed in a Pfizer release on Friday, “Many individuals coping with hemophilia B find it challenging to adhere to regular [factor IX] infusions, along with enduring spontaneous bleeding episodes, which can result in painful joint damage and mobility limitations.”
Cuker further emphasized that Pfizer’s drug “has the potential to be revolutionary for suitable patients by alleviating both the medical and treatment challenges in the long run.” This approval signifies a significant milestone for Pfizer, which is striving to recover from the sharp decline in its COVID-related business last year. The company is placing substantial bets on cancer medications and therapies for other disease domains to facilitate its business turnaround.
Pfizer is among numerous companies venturing into the swiftly expanding realm of gene and cell therapies, which involve one-time, high-cost treatments aimed at directly addressing a patient’s genetic source or cell to cure or substantially modify the progression of a disease. Certain health professionals anticipate these therapies will supplant traditional lifelong treatments typically used to manage chronic illnesses. In 2014, Pfizer acquired the rights to manufacture and market Beqvez from Spark Therapeutics.
According to a spokesperson speaking to CNBC, the company is introducing a warranty program to payers, covering patients undergoing treatment with Beqvez. Pfizer anticipates that this program will provide “financial security by offering insurance against the risk of efficacy failure,” as stated in the release. Beqvez will face competition from Hemgenix, a similar treatment by Australia-based CSL Behring, which received FDA approval for hemophilia B in 2022. Both drugs share a comparable list price of $3.5 million in the U.S. before insurance and other rebates.
Significantly, according to certain health experts, factors such as high costs and logistical challenges have constrained the adoption of Hemgenix and another approved gene therapy designed for the more prevalent hemophilia A. Additionally, Pfizer is pursuing FDA approval for its experimental antibody, marstacimab, for the treatment of hemophilia A and B. Moreover, the company is actively engaged in the development of a gene therapy targeting Duchenne muscular dystrophy, a genetic disorder characterized by progressive muscle weakening.
