The U.S. Food and Drug Administration has granted approval for a groundbreaking RNA-based treatment for hemophilia, marking a significant advancement in therapy 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, Qfitlia employs genetic code to inhibit the production of a protein that prevents blood cells from clumping together.
After a decade of human trials, the drug received FDA authorization based on the results of 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, regardless of whether patients had developed inhibitors – antibodies that can interfere with conventional replacement therapy.
The clinical trials, which included an extension study, revealed approximately 70% reduction in annual bleeding rates compared to control groups receiving standard clotting agent infusions. The drug will be administered as a subcutaneous injection every two months, with dosing adjusted according to companion diagnostic testing results.
The FDA has approved Qfitlia for routine prophylaxis in patients aged 12 and older with hemophilia A or B, including those with or without neutralizing antibodies. According to Brian Foard, who leads Sanofi’s specialty care division, the drug represents a potential paradigm shift in hemophilia treatment through its combination of effective bleeding prevention, reduced dosing frequency, and simplified administration method.
The medication emerged from a collaboration between Sanofi and Alnylam Pharmaceuticals, with Sanofi acquiring global rights to the drug in 2018 through a revised partnership agreement. Under this arrangement, Alnylam will receive royalties based on net sales.
Sanofi has set the annual list price at approximately $642,000, though the company expects the actual cost to be lower after accounting for discounts, rebates, and patient assistance programs. This pricing positions Qfitlia in line with other prophylactic treatments, 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 experiencing 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 field already well-served by effective treatments. The hemophilia community has shown some resistance to adopting innovative therapies, as evidenced by the slow uptake of gene therapies from companies like CSL Behring and BioMarin Pharmaceuticals. This cautious approach was further highlighted by Pfizer’s recent decision to discontinue its hemophilia B gene therapy, Beqvez, due to insufficient demand.
Dr. Guy Young from Children’s Hospital, Los Angeles, emphasized Qfitlia’s distinctive advantage of requiring the fewest doses among available prophylactic hemophilia treatments, potentially offering patients a more convenient therapeutic option in the evolving landscape of hemophilia care.