The United States Food and Drug Administration has granted approval for 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, fitusiran employs genetic code to inhibit the production of a protein that prevents blood cells from clumping together.
The drug’s approval follows a decade of human trials, with
authorization based on two pivotal late-stage clinical studies. These trials demonstrated the drug’s effectiveness in managing bleeding events in both major forms of hemophilia, showing positive results regardless of whether patients had developed inhibitors – antibodies that can interfere with conventional replacement therapy. The combined research, including an extension study, revealed approximately 70% reduction in annual bleeding rates compared to control groups.
Developed through a collaboration between Sanofi and Alnylam Pharmaceuticals, with Sanofi acquiring global rights in 2018, Qfitlia will be administered as a subcutaneous injection every two months. The treatment is approved for patients aged 12 and older with hemophilia A or B, including those with neutralizing antibodies. Dosing will be customized using a companion diagnostic test.
Brian Foard, who leads Sanofi’s specialty care division, emphasized the drug’s potential to revolutionize hemophilia treatment through its combination of effective bleeding prevention, extended dosing intervals, and simplified administration. Dr. Guy Young from Children’s Hospital, Los Angeles, highlighted that Qfitlia requires fewer doses than any other prophylactic therapy currently available for hemophilia.
The annual list price for Qfitlia has been set at approximately $642,000, though Sanofi anticipates lower actual costs after accounting for various discounts, rebates, and patient assistance programs. This pricing strategy places it in a similar range to 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 complimentary 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, including three existing 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 innovative 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.
The approval represents a significant milestone in hemophilia treatment, offering patients a new option with a unique mechanism of action and reduced dosing frequency. However, the success of Qfitlia will likely depend on how well Sanofi can navigate the complex landscape of patient preferences, existing treatment options, and access to care in the hemophilia market.