The U.S. Food and Drug Administration has granted approval to a groundbreaking RNA-based treatment for hemophilia, marking a significant advancement in therapeutic options for this rare bleeding disorder.
The newly approved medication, fitusiran, which will be marketed under the brand name Qfitlia by Sanofi, represents a novel approach to treating hemophilia. Unlike traditional treatments that focus on replacing or stimulating the production of missing blood-clotting proteins, this innovative therapy employs a different mechanism. It works by interfering with the production of a protein that prevents blood cells from clumping together.
Following a decade of human trials, the FDA’s approval was supported by data from two pivotal late-stage clinical studies. These trials demonstrated the drug’s effectiveness in managing bleeding events in patients with the two most prevalent forms of hemophilia. Notably, the treatment proved successful regardless of whether patients had developed inhibitors – antibodies that can compromise the
effectiveness of conventional replacement therapies.
The clinical trials revealed impressive results, with fitusiran reducing annual bleeding rates by approximately 70% compared to control groups. The drug will be administered as a subcutaneous injection every two months, with dosing adjusted according to results from a companion diagnostic test.
Sanofi, which collaborated with Alnylam Pharmaceuticals in developing the drug, obtained global rights to fitusiran through a partnership agreement in 2018. Under this arrangement, Alnylam will receive royalties based on the drug’s net sales.
The FDA has approved Qfitlia for routine prophylaxis in individuals aged 12 and older with either hemophilia A or B, including those with neutralizing antibodies. Dr. Guy Young, who directs the Hemostasis and Thrombosis Center at Children’s Hospital, Los Angeles, emphasized that the drug offers the least frequent dosing schedule among all prophylactic hemophilia treatments.
Sanofi has set the annual list price for Qfitlia at approximately $642,000, though actual costs are expected to be lower after accounting for various discounts, rebates, and patient assistance programs. This pricing is comparable to other prophylactic treatments in the market, such as Roche’s Hemlibra, which launched in 2017 with a list price exceeding $480,000.
To facilitate patient access, Sanofi has implemented several support programs, including six months of free medication for first-time patients while awaiting insurance coverage decisions. The company is also offering co-pay assistance and bridge programs for patients facing insurance coverage gaps or other payer-related challenges.
Despite Sanofi’s established presence in the rare disease market and its existing portfolio of three hemophilia products, the company may face challenges in marketing Qfitlia. The hemophilia treatment landscape is already well-populated with effective therapies, and the patient community has shown some resistance to adopting new treatment modalities. This hesitancy is evidenced by the slow uptake of gene therapies from companies like CSL Behring and BioMarin
Pharmaceuticals, and Pfizer’s recent decision to discontinue its hemophilia B gene therapy, Beqvez, due to insufficient demand.
Brian Foard, who leads Sanofi’s specialty care division, expressed optimism about the drug’s potential impact, suggesting that Qfitlia could significantly transform hemophilia treatment through its combination of effective bleeding prevention, infrequent dosing requirements, and simplified administration method.