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.
Updated statement from Genentech on Hemlibra, March 28, 2018.
The screening kit was developed for broad utility for both laboratories seeking a standardized inhibitor assay for clinical management and multi-center clinical studies of patients with hemophilia A
NHF received information from Genentech’s Clinical Development Lead regarding reports of the death of patients utilizing Hemlibra (emicizumab).
Aptevo Therapeutics recently announced patient-reported data on individuals currently being treated with IXINITY®, the company’s recombinant factor IX product.
eNewsletter Sign Up
Steps for Living
Victory for Women
Unite for Bleeding Disorders Walk
Donate Your Car