Sanofi/Alnylam's siRNA Therapy Qfitlia™ (Fitusiran) Receives FDA Approval for Hemophilia A and B

On March 28, Alnylam announced that the U.S. FDA approved Qfitlia™ (fitusiran) for marketing. This is the sixth RNAi therapeutic drug discovered by Alnylam to be approved in the United States and the first and only treatment that reduces antithrombin (AT), a protein that inhibits blood clotting, with the aim of promoting thrombin generation to rebalance hemostasis and prevent bleeding. Qfitlia is used in the United States for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients aged 12 years and older with Hemophilia A or B, with or without Factor VIII or IX inhibitors (neutralizing antibodies). Alnylam's scientists discovered Qfitlia and published the first clinical data in The New England Journal of Medicine in 2017, showing a reduction in bleeding rates among hemophilia patients, and initiated a Phase 3 clinical development program. In 2014, Sanofi obtained global rights to co-develop and co-commercialize Qfitlia under a license and collaboration agreement. The agreement was revised in 2018, with Sanofi obtaining global development and commercialization rights to Qfitlia, and Alnylam being eligible to receive tiered royalties ranging from 15% to 30% on global net sales.Qfitlia is a new siRNA drug that utilizes Alnylam's ESC-GalNAc conjugation technology.

Qfitlia is the sixth drug discovered by Alnylam using its RNAi therapeutic platform to be approved so far. This approval marks the completion of the "product" goal in Alnylam's P5x25 strategy, a set of corporate objectives for 2025. Qfitlia has the potential to benefit approximately 1 million patients worldwide with Hemophilia A and B.

Alnylam CEO Yvonne Greenstreet stated:"Today's approval of Qfitlia is an important moment for Alnylam, our RNAi therapeutics platform, and hemophilia patients, who now have access to what we believe will be a transformative drug. Clinical studies have shown that by reducing antithrombin, Qfitlia can preventively reduce the annual bleeding rate by 90%. Importantly, based on its unique mechanism of action, Qfitria has demonstrated efficacy in patients with hemophilia A or B, with or without inhibitors, administered subcutaneously once every two months. We are excited about the potential of Qfitlia to become a significant non-factor option for patients with this burdensome disease."

Qfitlia is used in the United States to treat hemophilia A or B in adult and pediatric patients 12 years of age and older, with or without inhibitors to factor VIII (hemophilia A) or factor IX (hemophilia B). Regulatory submissions for Qfitlia have also been completed in China and Brazil.