Sarepta Therapeutics will restart shipments of its gene therapy after US regulators gave clearance to resume limited distribution. This decision came after a brief pause requested by the Food and Drug Administration. The company halted shipments following the deaths of two teenage patients. These teens had received the therapy for Duchenne muscular dystrophy. Later, the death of an 8-year-old boy raised additional alarm.
However, after further review, the FDA confirmed the young boy’s death was unrelated to the gene therapy. This decision allowed Sarepta to resume gene therapy shipments for certain patients. Specifically, the FDA approved shipments for boys who still retain the ability to walk. This subgroup has shown better response to the therapy. The clearance to resume gene therapy shipments significantly improves Sarepta’s commercial outlook.
The therapy, called Elevidys, treats Duchenne muscular dystrophy, a fatal disease that leads to severe muscle wasting. The disease mainly affects young boys and often results in early death. Elevidys first received accelerated approval for walking patients. It was later approved for older patients, including those who had lost the ability to walk. However, safety concerns caused regulators to act quickly.
Following the deaths from liver complications, the FDA called for a pause. Sarepta had to halt new distributions of the product to reassess risks. Meanwhile, investors and analysts watched closely. The latest FDA statement confirmed that non-walking patients remain under a voluntary hold. The agency asked Sarepta to provide more safety data before allowing wider use.
Market reaction to the FDA’s decision was strong. Sarepta shares rose more than 16% in after-hours trading. Investors welcomed the news, expecting stronger future sales for Elevidys. Analysts believe this approval helps restore confidence in the company’s key therapy. They expect Elevidys to perform well in the short term. The return of shipments could drive meaningful revenue growth.
Nevertheless, the company still faces regulatory hurdles. It must conduct more studies to ensure safety, especially for non-walking patients. The FDA continues to monitor all ongoing cases carefully. Despite the risks, the restart signals optimism for both the company and families hoping for effective treatments. Sarepta plans to work closely with the FDA to expand access.
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