Hot topic: FDA’s ‘plausible mechanism’ pathway could boost cell and gene therapies
Good news for developers of drugs for ultra-rare diseases – including cell and gene therapy firms – in the form of a new way of assessing them that’s being proposed by the US’s Food and Drug Administration (FDA). The regulator is looking at introducing what it calls a “plausible mechanism” pathway. This would allow approval of medicines for ultra-rare diseases, if there are very […]