Timeline of the gene therapy’s clinical development and regulatory review shows agency staff repeatedly questioned the ability of micro-dystrophin to predict clinical benefit in Duchenne muscular dystrophy patients.
Throughout Sarepta Therapeutics, Inc.’s clinical development of the gene therapy Elevidys (delandistrogene moxeparvovec-rokl, formerly SRP-9001), US Food and Drug Administration review staff repeatedly questioned the ability of micro-dystrophin, a surrogate endpoint, to predict clinical benefit in Duchenne muscular dystrophy patients.
Review documents show that in at least four separate meetings with Sarepta dating back to December 2018, Center for Biologics...
The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.
Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.
The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.
The Pink Sheet drug review profile investigates the long and rigorous process behind the FDA’s recent relaxation of the cardiovascular safety warning for testosterone products.
The agency said it will consider knee extensor muscle strength as a potential intermediate clinical endpoint to support accelerated approval, but Stealth CEO Reenie McCarthy said announcing it via complete response letter is confusing and inefficient.
The European Medicines Agency says that a regulatory reliance pilot that it is supporting to speed up assessments of post-approval changes appears to be accelerating evaluation timelines in non-EU countries.
The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include two new products, one of which is Vyjuvek, Krystal Biotech's advanced therapy for treating dystrophic epidermolysis bullosa.
