The first stage will collect data that will be eventually used to evaluate the efficacy and safety of the investigational hemophilia B therapy.
The update reported on a Phase 1/2 clinical trial for the company’s investigational therapy for the treatment of patients with severe hemophilia B/factor IX (FIX) deficiency.
This priority review is specifically for adults and children with hemophilia A without FVIII inhibitors.
The new vial size is 3500 IU (international unit).
The data showed its hemophilia B investigational gene therapy candidate AMT-060 could be successfully delivered to patients with certain pre-existing antibodies.
The overall annualized bleeding rate for all 15 participants was reduced by 98% to an annual rate of 0.2 bleeds per patient.
In the Phase III HAVEN 3 study, patients who received HEMLIBRA prophylaxis every week or every two weeks experienced a 96% and 97% reduction in treated bleeds.
The designation is for people with hemophilia A without inhibitors.
ICER is an independent and non-partisan research organization that evaluates the clinical and economic value of prescription drugs, medical tests, and other healthcare and healthcare delivery innovations.
The company donated over 25 million international units (IU) of factor products used to treat hemophilia A and B.
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