The US Food and Drug Administration (FDA) has granted approval for Qfitlia (fitusiran), an RNA interference (RNAi) therapeutic developed by Alnylam Pharmaceuticals, for the treatment of haemophilia A and B.
Qfitlia is the first therapeutic agent aimed at reducing antithrombin levels, which inhibits blood clotting, to promote thrombin generation and restore haemostatic balance.
It is indicated for routine prophylaxis in adults and children aged 12 and older with haemophilia A or B, irrespective of the presence of factor VIII or IX inhibitors.
This approval represents the sixth RNAi therapeutic from Alnylam Pharmaceuticals to secure approval in the US. The milestone aligns with the company’s P5x25 strategic plan to bring multiple RNAi therapeutics to market by 2025.
Alnylam Pharmaceuticals’ clinical data, first published in 2017 in the New England Journal of Medicine, demonstrated Qfitlia’s efficacy in reducing bleeding rates among haemophilia patients.
Alnylam Pharmaceuticals CEO Yvonne Greenstreet said: “Clinical studies showed that by lowering antithrombin, Qfitlia was able to prophylactically reduce annualised bleeding rates by 90%.
“Importantly, based on its unique mechanism of action, Qfitlia has demonstrated efficacy in patients with haemophilia A or B, with or without inhibitors, with a subcutaneous injection regimen once every two months.”
In 2014, Sanofi obtained global rights to co-develop and commercialise the asset under a licensing agreement. In 2018, the agreement was modified, granting Sanofi full global development and commercialisation rights.
Alnylam Pharmaceuticals will receive tiered royalties ranging from 15% to 30% based on worldwide net sales.
Qfitlia also awaits regulatory approval in China and Brazil.
With an estimated one million individuals affected by haemophilia A and B worldwide, Qfitlia offers a potential new treatment option for this population.
Concurrently, Alnylam Pharmaceuticals has announced that the FDA has approved a supplemental new drug application for AMVUTTRA (vutrisiran). This approval expands AMVUTTRA’s use to treat cardiomyopathy associated with wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults.
The expanded indication aims to reduce cardiovascular mortality and hospitalisations, establishing AMVUTTRA as the first FDA-approved treatment for both ATTR-CM and hereditary transthyretin-mediated amyloidosis polyneuropathy (hATTR-PN) in adults.
