01 Jun FDA wishes faster approval for gene therapies
FDA wishes to speed approval for new therapies aimed at diseases caused by defective genes.
Gene therapies are meant to be used only once for “devastating diseases, many of which are fatal and lack available therapy”, which leads us to “accept more uncertainty and facilitate timely access to these promising therapies” says FDA Commissioner Scott Gottlieb.
In order to keep pace with one of biotechnology’s fastest-growing fields, the FDA could deliver its approval based on a biological or chemical measure of the drug’s effectiveness, instead of whether it actually cures the disease.
In other words, the US regulatory body would base their decision on surrogate endpoints as opposed to traditional clinical endpoints that could be confirmed post-approval, thus allowing a faster access to these drugs and reducing the extraordinarily expensive manufacturing costs of these long-awaited therapies.
The agency plans to focus first on hemophilia and could approve the gene therapies in development based on how much clotting factor the patient produces after treatment, instead of the number of bleeds he or she will face.
This new path may change the prospect of more than 500 experimental drugs now in development.