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Boston Globe
29-07-2025
- Business
- Boston Globe
Sarepta soars after FDA says some patients can get Elevidys
Sarepta shares rose 14 percent Tuesday, placing the company among the biggest gainers in the Nasdaq Composite index at that point. On Monday, the shares gained 16 percent ahead of the FDA's announcement. Through Monday's close, the company's stock had fallen 89 percent year to date. Get Starting Point A guide through the most important stories of the morning, delivered Monday through Friday. Enter Email Sign Up The FDA decision was a relief to Sarepta investors who were worried about the company's future since it owes money to a biotech partner and faces $1.2 billion of debt due in 2027. Advertisement Sarepta must make a $100 million milestone payment to Arrowhead Pharmaceuticals Inc. within the next two months as part of a deal between the two companies. After the FDA asked Sarepta to halt sales of Elevidys this month, its debt began trading at a level that indicated investors questioned the company's ability to repay it. Advertisement The FDA's move 'significantly improves Elevidys' sales outlook in the near-term,' Jefferies analyst Andrew Tsai said in a note. RBC Capital Markets analyst Brian Abrahams said the move 'takes the edge off solvency concerns to some extent' for the company, but won't eliminate its challenges. The drug 'will likely be used less than previously estimated,' he wrote, adding it was likely to achieve no more than $300 million in peak sales for walking patients. Two teenage boys died of acute liver failure in recent months after taking Elevidys, which is used to treat Duchenne muscular dystrophy. They weren't able to walk because of the muscle-wasting disease. The company also has said that a 51-year-old patient died of acute liver failure last month in an early-stage trial of a gene therapy that works in a similar way to Elevidys. After the first two deaths, Sarepta stopped shipping the drug for nonwalking, older patients. The drug remains paused for these people, though the FDA said it would also work with Sarepta regarding those patients. In a statement, Sarepta said it will resume shipping of Elevidys 'imminently.' 'We remain committed to working closely with the FDA to ensure that all decisions are grounded in science and the best interests of patients, considering the compelling need of these families to access disease-modifying therapy,' the company said. The FDA also said it had concluded that the death of an 8-year-old in Brazil was not related to the therapy. It had said it was investigating the death late Friday. The FDA's decision to make Elevidys available again was praised by patient advocates. In a statement, Debra Miller, founder of CureDuchenne, said many families facing the muscle disorder 'have expressed that they understand the risks of gene therapy, and they deserve the opportunity to make their own informed decisions.' Advertisement
29-07-2025
- Business
Sarepta will resume gene therapy shipments after FDA review of recent patient death
WASHINGTON -- Shares of beleaguered drugmaker Sarepta Therapeutics jumped in afterhours trading Monday after the company said it would resume shipping its gene therapy for some patients, following a brief pause requested by regulators. The Food and Drug Administration said it recommended lifting the hold for young patients with Duchenne's muscular dystrophy who are still able to walk. Regulators had requested the pause after the deaths of two older teenagers who were taking the therapy. The FDA also said in a statement it determined that a recently reported death of an 8-year-old boy was unrelated to the therapy. Company shares surged more than 16% after the announcement to $13.86 in afterhours trading. The jump is the latest in a series of drastic stock movements triggered by changing fortunes for the company's best-selling product. Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, the fatal muscle-wasting disease that affects boys and young men, resulting in early death. It received accelerated approval in 2023 for a narrow range of young patients and was expanded last year for use in older patients, including those who can no longer walk. The FDA decision Monday 'significantly improves Elevidys' sales outlook in the near-term,' Jefferies analyst Andrew Tsai told investors, in a note after the announcement. 'The street will feel relieved about the situation, suggesting meaningful stock upside potential.' Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. The FDA then requested a pause in shipments of the drug after the death of a third patient taking a different Sarepta therapy. FDA officials have suggested the company will need to provide new study data on safety to resume Elevidys' use in older patients. 'The FDA will continue to work with the sponsor regarding non-ambulatory patients, which remains subject to a voluntary hold, following two deaths,' FDA said in its statement. ___ The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
The Hill
28-07-2025
- Business
- The Hill
Sarepta will resume gene therapy shipments after FDA review of recent patient death
WASHINGTON (AP) — Shares of beleaguered drugmaker Sarepta Therapeutics jumped in afterhours trading Monday after the company said it would resume shipping its gene therapy for some patients, following a brief pause requested by regulators. The Food and Drug Administration said it recommended lifting the hold for young patients with Duchenne's muscular dystrophy who are still able to walk. Regulators had requested the pause after the deaths of two older teenagers who were taking the therapy. The FDA also said in a statement it determined that a recently reported death of an 8-year-old boy was unrelated to the therapy. Company shares surged more than 16% after the announcement to $13.86 in afterhours trading. The jump is the latest in a series of drastic stock movements triggered by changing fortunes for the company's best-selling product. Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, the fatal muscle-wasting disease that affects boys and young men, resulting in early death. It received accelerated approval in 2023 for a narrow range of young patients and was expanded last year for use in older patients, including those who can no longer walk. The FDA decision Monday 'significantly improves Elevidys' sales outlook in the near-term,' Jefferies analyst Andrew Tsai told investors, in a note after the announcement. 'The street will feel relieved about the situation, suggesting meaningful stock upside potential.' Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. The FDA then requested a pause in shipments of the drug after the death of a third patient taking a different Sarepta therapy. FDA officials have suggested the company will need to provide new study data on safety to resume Elevidys' use in older patients. 'The FDA will continue to work with the sponsor regarding non-ambulatory patients, which remains subject to a voluntary hold, following two deaths,' FDA said in its statement. ___ The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
San Francisco Chronicle
28-07-2025
- Business
- San Francisco Chronicle
Sarepta will resume gene therapy shipments after FDA review of recent patient death
WASHINGTON (AP) — Shares of beleaguered drugmaker Sarepta Therapeutics jumped in afterhours trading Monday after the company said it would resume shipping its gene therapy for some patients, following a brief pause requested by regulators. The Food and Drug Administration said it recommended lifting the hold for young patients with Duchenne's muscular dystrophy who are still able to walk. Regulators had requested the pause after the deaths of two older teenagers who were taking the therapy. The FDA also said in a statement it determined that a recently reported death of an 8-year-old boy was unrelated to the therapy. Company shares surged more than 16% after the announcement to $13.86 in afterhours trading. The jump is the latest in a series of drastic stock movements triggered by changing fortunes for the company's best-selling product. Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, the fatal muscle-wasting disease that affects boys and young men, resulting in early death. It received accelerated approval in 2023 for a narrow range of young patients and was expanded last year for use in older patients, including those who can no longer walk. The FDA decision Monday 'significantly improves Elevidys' sales outlook in the near-term,' Jefferies analyst Andrew Tsai told investors, in a note after the announcement. 'The street will feel relieved about the situation, suggesting meaningful stock upside potential.' Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. The FDA then requested a pause in shipments of the drug after the death of a third patient taking a different Sarepta therapy. FDA officials have suggested the company will need to provide new study data on safety to resume Elevidys' use in older patients. 'The FDA will continue to work with the sponsor regarding non-ambulatory patients, which remains subject to a voluntary hold, following two deaths,' FDA said in its statement. ___ The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
Winnipeg Free Press
28-07-2025
- Business
- Winnipeg Free Press
Sarepta will resume gene therapy shipments after FDA review of recent patient death
WASHINGTON (AP) — Shares of beleaguered drugmaker Sarepta Therapeutics jumped in afterhours trading Monday after the company said it would resume shipping its gene therapy for some patients, following a brief pause requested by regulators. The Food and Drug Administration said it recommended lifting the hold for young patients with Duchenne's muscular dystrophy who are still able to walk. Regulators had requested the pause after the deaths of two older teenagers who were taking the therapy. The FDA also said in a statement it determined that a recently reported death of an 8-year-old boy was unrelated to the therapy. Company shares surged more than 16% after the announcement to $13.86 in afterhours trading. The jump is the latest in a series of drastic stock movements triggered by changing fortunes for the company's best-selling product. Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, the fatal muscle-wasting disease that affects boys and young men, resulting in early death. It received accelerated approval in 2023 for a narrow range of young patients and was expanded last year for use in older patients, including those who can no longer walk. The FDA decision Monday 'significantly improves Elevidys' sales outlook in the near-term,' Jefferies analyst Andrew Tsai told investors, in a note after the announcement. 'The street will feel relieved about the situation, suggesting meaningful stock upside potential.' Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. The FDA then requested a pause in shipments of the drug after the death of a third patient taking a different Sarepta therapy. FDA officials have suggested the company will need to provide new study data on safety to resume Elevidys' use in older patients. 'The FDA will continue to work with the sponsor regarding non-ambulatory patients, which remains subject to a voluntary hold, following two deaths,' FDA said in its statement. ___ The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
