Sarepta must perform enhanced pharmacovigilance for serious renal toxicity events and rhabdomyolysis with the Duchenne muscular dystrophy drug, which carries a label warning for renal toxicity. Approval letter includes 2024 target completion date for ESSENCE confirmatory trial.
The US Food and Drug Administration is relying on enhanced pharmacovigilance and a label warning to address one of the safety concerns that Sarepta Therapeutics Inc. said delayed approval of its Duchenne muscular dystrophy drug Vyondys 53 (golodirsen).
Golodirsen received accelerated approval 12 December for patients with a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. The approval was a surprise, coming less...
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 UK regulator’s draft guideline on the use of external control arms based on real-world data reflects concepts similar to those outlined by the US Food and Drug Administration.
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.
