Sarepta Shares Bounce As Pfizer’s DMD Gene Therapy Sparks Safety Concerns
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.
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Updated Phase Ib data showed safety issues due to complement activation, but Pfizer is confident the benefits outweigh the risks and monitoring systems will help as it moves into Phase III.
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.
The US FDA issued a complete response letter rejecting golodirsen based on risk of infection at intravenous infusion ports and preclinical renal toxicity.