FDA Approves Two Alnylam RNAi Therapies

Alnylam delivered a one-two punch in March, growing its portfolio of FDA-approved RNAi therapies with the addition of Amvuttra (vutrisiran) for transthyretin amyloid cardiomyopathy (ATTR-CM) and Qfitlia (fitusiran) for hemophilia.

Qfitlia (fitusiran) is the sixth Alnylam-discovered RNAi therapeutic approved in the U.S., and the first and only therapeutic to lower antithrombin (AT), a protein that inhibits blood clotting, and is indicated in the U.S. to prevent or reduce the frequency of bleeding episodes in hemophilia A or B. Regulatory submissions for Qfitlia have also been completed in China and Brazil.

Alnylam scientists discovered Qfitlia, published the first clinical data in 2017 and initiated the Phase 3 development program. In 2014, Sanofi obtained global rights to co-develop and co-commercialize Qfitlia under a license and collaboration agreement. The agreement was amended in 2018, with Sanofi obtaining global development and commercialization rights to Qfitlia, and Alnylam becoming eligible to receive tiered royalties of 15-30 percent on net sales.

Earlier in March, Alnylam won FDA approval for a second RNAi therapeutic, Amvuttra (vutrisiran), for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM), a rapidly progressive and ultimately fatal heart ailment estimated to affect approximately 150,000 people in the U.S. and over 300,000 people worldwide.

There is no cure for ATTR-CM which is characterized by the deposition of misfolded transthyretin (TTR) fibrils which form amyloid and cause irreversible cardiovascular damage and premature death. Amvuttra works upstream, addressing the disease at its source by rapidly knocking down TTR production and thereby substantially decreasing deposition of TTR fibrils.

Marketing authorization applications for Amvuttra are currently under review by several global health agencies including the European Medicines Agency and the Japanese Pharmaceuticals and Medical Device Agency. In addition, Alnylam remains on track to proceed with further global regulatory submissions for vutrisiran in 2025. (Source: Alnylam Website, 20 & 28 March, 2025)