Hemophilia

The phase 3 trial of valoctocogene roxaparvovec for severe hemophilia A showed it reduced bleeding events as well as the use of factor VIII concentrates.

The company, uniQure, said it is the largest group of patients with hemophilia B to receive a single gene therapy investigational product so far. 

Up until now, it was thought most patients with hemophilia A developed inhibitors in middle-age. A new study suggests it usually happens in the early teens.

Hemophilia care principles have been on the rise in Europe over the course of the last decade, however, differing regions and policies on the continent reveal gaps in access to various treatments and specialty services.

The meta-analysis showed that more than 1,125,000 men around the world have the inherited bleeding disorder; 418,000 of those have a severe version of the mostly undiagnosed disease. Previously, only 400,000 people globally were estimated to have hemophilia.