The results are from the prospective, multicenter, phase 3 explorer7 trial, involving 133 patients, including 80 with hemophilia A and 53 had hemophilia B.
1. Annualized bleeding rate was reduced by 91% in the fitusiran prophylaxis group compared to the bypassing agents on-demand group. 2. 5% of patients in the fitusiran group reported thromboembolic events; there were no deaths. Evidence Rating Level: 1 (Excellent) Study Rundown: Hemophilia is marked by a deficiency in certain coagulation factors that make patients
By selling partial royalties to approved hemophilia gene therapy Hemgenix, uniQure gains immediate cash to support its pipeline, including a Huntington’s disease gene therapy already in the clinic . The royalty deal extends uniQure’s cash runway into 2026.
Specific dosing regimens of treatments prescribed by blood specialists have not been widely studied, despite the advent of newer hemophilia therapies, the researchers said.
Despite an anticipated price tag of $2.5 million, there s no way to predict who will have long-standing benefit from valoctocogene roxaparvovec and who will not.