It is the dawn of gene therapy in DMD and the age of Elevidys [aka SRP-9001].
There is nonetheless a certain muted celebration due to the fact that the approval is based upon a surrogate endpoint i.e. an increase in micro-dystrophin protein expression; Elevidys produces a biological effect rather than an improvement in symptoms. Further, the drug is restricted to the treatment of ambulatory pediatric patients aged 4 years through 5 years which again raises the question why does turning six suck.
Its informative to review the press release by PPMD. Firstly, PPMD applauds the FDA for the landmark approval of the first ever gene therapy treatment but additionally, the advocacy group acknowledges that it will take a combination of therapies to fully halt the progression of Duchenne.
The accelerated approval process is now out of the way. Arguably, the focus will now shift towards combination therapies that will turn biological effects into actual improvement in symptoms.
