Posted by Michael Wonder on 23 May 2018
Gene therapies that could transform diseases get easier FDA path
23 May 2018 - Haemophilia treatments could be first under proposed method.
New therapies that may cure diseases caused by defective genes will get a faster path to approval by U.S. regulators, part of an effort by the FDA to keep pace with one of biotechnology’s fastest-growing fields.
Unlike traditional drugs, gene therapies are intended to be given once, transform the inner workings of the body and last for a lifetime. For regulators, the challenge is to find ways to get the new therapies to desperate patients while balancing the need to monitor their long-term safety for years after approval, FDA Commissioner Scott Gottlieb said Tuesday.
