Alnylam Pharma Gains Approval for Qfitlia™ (Fitusiran) for Hemophilia

Alnylam Pharmaceuticals has received approval from the United States Food and Drug Administration (FDA) for Qfitlia™ (fitusiran), the first small interfering RNA (siRNA) therapy for treating haemophilia A or B.

RNA interference (RNAi) is a natural cellular process that silences specific genes and is recognised as a significant advancement in biology and drug development. The discovery of RNAi was awarded the Nobel Prize in Physiology or Medicine in 2006. 

RNAi therapeutics utilise small interfering RNA (siRNA) molecules to silence messenger RNA (mRNA), preventing the production of disease-related proteins. This approach offers a novel method for treating genetic and other diseases, with the potential to significantly impact patient care.

Qfitlia is the sixth RNA interference (RNAi) therapeutic discovered by the company to receive regulatory approval in the United States. It is designed to lower antithrombin, a protein that inhibits blood clotting, to support thrombin generation, rebalance haemostasis, and prevent bleeding episodes.

Qfitlia is indicated for routine prophylaxis to reduce the frequency of bleeding episodes in adults and children aged 12 years and older with haemophilia A or B, including those with or without factor VIII or IX inhibitors. 

The therapy was initially developed by Alnylam, with clinical data first published in 2017 demonstrating its effectiveness in reducing bleeding rates. Sanofi acquired the global development and commercialisation rights in 2018 under a revised collaboration agreement, with Alnylam entitled to receive tiered royalties on global sales.

This approval represents a key milestone in Alnylam’s corporate objectives and is expected to benefit a significant number of individuals living with haemophilia A and B worldwide. Clinical studies have shown that by reducing antithrombin levels, Qfitlia can lower annual bleeding rates by 90%. 

The therapy is administered via subcutaneous injection once every two months and offers a non-factor treatment option for patients.

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