The U.S. Food and Drug Administration has given its 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, operates through a novel mechanism distinct from traditional hemophilia treatments. While existing therapies typically focus on replacing or stimulating the production of missing blood-clotting proteins, this innovative drug takes a different approach by interfering with the production of a protein that prevents blood cells from clumping together.
After 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, showing positive results regardless of whether patients had developed inhibitors – antibodies that can compromise the effectiveness of conventional replacement therapies.
The clinical trials, which included an extension study, revealed impressive results, with the drug reducing annual bleeding rates by approximately 70% compared to control groups receiving standard clotting agents. The medication will be administered as a subcutaneous injection every two months, with dosing regulated through a companion diagnostic test.
Sanofi, which partnered with Alnylam Pharmaceuticals to develop the drug, gained worldwide rights to fitusiran in 2018 through a revised collaboration agreement. Under this arrangement, Alnylam will receive royalties based on the drug’s net sales.
The FDA has authorized Qfitlia for routine prophylactic treatment in individuals aged 12 and older with either hemophilia A or B, including those with neutralizing antibodies. Brian Foard, who leads Sanofi’s specialty care division, emphasized the drug’s potential to
revolutionize hemophilia treatment through its combination of effective bleeding protection and convenient dosing schedule.
Dr. Guy Young, director of the Hemostasis and Thrombosis Center at Children’s Hospital, Los Angeles, highlighted that Qfitlia offers the most infrequent dosing schedule among all prophylactic hemophilia treatments currently available.
The drug’s annual list price has been set at approximately $642,000, though Sanofi anticipates the actual cost will be lower once various discounts, rebates, and patient assistance programs are applied. 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 insurance coverage is being determined. The company is also offering co-pay assistance and bridging support 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
commercialization of Qfitlia may face challenges in a market already well-served by effective treatments. The hemophilia community has shown some reluctance to embrace cutting-edge therapies, as evidenced by the slow adoption of gene therapies from companies like CSL Behring and BioMarin Pharmaceuticals. This hesitancy was further highlighted by Pfizer’s recent decision to discontinue its hemophilia B gene therapy, Beqvez, due to insufficient demand.