Qfitlia™: Mechanism of Action and Clinical Benefits#

Qfitlia™'s novel mechanism of action sets it apart from traditional hemophilia treatments. By reducing AT levels, Qfitlia™ facilitates thrombin generation, which is crucial for effective blood clotting. This approach offers a new way to manage hemophilia, irrespective of the presence of factor VIII or IX inhibitor antibodies. The drug, commercialized by Sanofi under a collaborative agreement with ALNY, is priced at approximately $642,000 per year. This high price reflects the innovative nature of the therapy and its potential to improve the lives of patients with hemophilia.

Projected Revenue Impact of Qfitlia™#

The approval of Qfitlia™ is projected to significantly boost ALNY's revenue. As the first siRNA therapeutic for hemophilia, Qfitlia™ is poised to capture a substantial market share. Alnylam will receive tiered royalties of 15-30% on global net sales of Qfitlia™, contributing to a steady revenue stream. Analysts estimate peak Qfitlia™ sales to reach just above $700 million in the US by 2030, and roughly $1.3 billion when accounting for EU markets (William Blair.

ALNY's overall revenue guidance for 2025 is $2.05-$2.25 billion, representing +31% growth. While the exact contribution of Qfitlia™ to this growth remains unspecified for the 2025-2027 timeframe, its long-term revenue potential is substantial. The drug's high price is justified by its innovative nature and the value it offers to patients. The convenience and efficacy of Qfitlia™ may position it as a preferred treatment option for many patients, despite its cost.

Vutrisiran's HELIOS-B Data: A Game Changer for ATTR-CM#

New data from the HELIOS-B Phase 3 clinical trial of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM) was presented at the American College of Cardiology's Annual Scientific Session (ACC.25). The HELIOS-B study evaluated vutrisiran for the treatment of ATTR-CM in a population representative of today's patients (American College of Cardiology.