Key clinical point: The RNA interference drug fitusiran appears to be safe and efficacious in hemophilia A and B.
Major finding: Once-monthly fitusiran therapy was associated with a reduction in median annualized bleeding rates from 42.00 to 1.04 in patients with inhibitors.
Study details: A phase 2, open-label extension study in 34 individuals with hemophilia A and B, with and without inhibitors, who were treated with fitusiran for at least 2 years.
Disclosures: The study was funded by Sanofi Genzyme and Alnylam Pharmaceuticals, and six authors were employees of Sanofi Genzyme. Dr. Pasi reported financial relationships with pharmaceutical companies, including Alnylam.
Pasi J et al. 2019 ISTH Congress, Abstract OC 11.3.