Sarepta Surprised By CRL For Exon 53-Skipping DMD Drug Golodirsen
The US FDA issued a complete response letter rejecting golodirsen based on risk of infection at intravenous infusion ports and preclinical renal toxicity.
You may also be interested in...
The US FDA has for the second time placed a clinical hold on Solid Biosciences’ potential gene therapy for Duchenne muscular dystrophy, a move linked to a 70% decline in the company’s share price. The company had reported a serious adverse event in a patient treated with higher doses of the viral vector-delivered gene therapy.
Pfizer is forging ahead with a Phase III trial for its DMD therapy, even though Phase Ib data raised safety worries. Gene therapy rival Sarepta’s shares were up by 17% on the news.
Sarepta Therapeutics has wasted no time in buying out partner Myonexus, swallowing up five extra clinical candidates, after studies of its lead gene therapy for limb-girdle muscular dystrophy showed positive results.