Latest news with #RituBaral
22-07-2025
- Business
Sarepta shares slide again as drugmaker bows to FDA pressure to pause gene therapy
WASHINGTON -- Shares of drugmaker Sarepta Therapeutics continued to sink Tuesday after the company said it would comply with a Food and Drug Administration request to pause shipments of its gene therapy following several patient deaths. The decision, announced late Monday, comes just days after the company rebuffed FDA regulators in an extremely unusual decision that alarmed investors and analysts. Sarepta CEO Doug Ingram said the company seeks a 'productive and positive' relationship with FDA and that 'maintaining that productive working relationship required this temporary suspension.' The Cambridge, Massachusetts-based company said it would ' temporarily pause all shipments' of its gene therapy Elevidys for muscular dystrophy at the close of business Tuesday. It's the latest in a series of highly irregular moves that have rocked company shares for weeks and forced it to lay off hundreds of staffers. 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. The one-time treatment was initially approved for boys age 4 and younger who could still walk. Last year, FDA expanded approval to older patients who are no longer able to walk. The therapy was already under FDA scrutiny after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. Then the company last week disclosed a third death with a different therapy: a 51-year-old patient who was enrolled in a company trial for another form of muscular dystrophy. FDA responded on Friday by asking the company to immediately halt all shipments of Elevidys. Company executives initially refused, noting that the latest death was not tied to Elevidys, its best-selling product. Wall Street analysts said the company made the right decision to cooperate. Defying the FDA would have 'irreparably damaged the company's relationship with FDA under the current leadership and administration,' TD Cowen analyst Ritu Baral told investors in a note Tuesday. Baral estimated the pause in distribution would last three to six months. The FDA has the authority to pull drugs from the market, but the process can take months or even years. Instead, the agency usually makes an informal request and companies almost always comply. Even in the rare cases when drugmakers haven't cooperated, the FDA has prevailed after public hearings and appeals. ___ 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
22-07-2025
- Business
- The Hill
Sarepta shares slide again as drugmaker bows to FDA pressure to pause gene therapy
WASHINGTON (AP) — Shares of drugmaker Sarepta Therapeutics continued to sink Tuesday after the company said it would comply with a Food and Drug Administration request to pause shipments of its gene therapy following several patient deaths. The decision, announced late Monday, comes just days after the company rebuffed FDA regulators in an extremely unusual decision that alarmed investors and analysts. Sarepta CEO Doug Ingram said the company seeks a 'productive and positive' relationship with FDA and that 'maintaining that productive working relationship required this temporary suspension.' The Cambridge, Massachusetts-based company said it would ' temporarily pause all shipments' of its gene therapy Elevidys for muscular dystrophy at the close of business Tuesday. It's the latest in a series of highly irregular moves that have rocked company shares for weeks and forced it to lay off hundreds of staffers. 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. The one-time treatment was initially approved for boys age 4 and younger who could still walk. Last year, FDA expanded approval to older patients who are no longer able to walk. The therapy was already under FDA scrutiny after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. Then the company last week disclosed a third death with a different therapy: a 51-year-old patient who was enrolled in a company trial for another form of muscular dystrophy. FDA responded on Friday by asking the company to immediately halt all shipments of Elevidys. Company executives initially refused, noting that the latest death was not tied to Elevidys, its best-selling product. Wall Street analysts said the company made the right decision to cooperate. Defying the FDA would have 'irreparably damaged the company's relationship with FDA under the current leadership and administration,' TD Cowen analyst Ritu Baral told investors in a note Tuesday. Baral estimated the pause in distribution would last three to six months. The FDA has the authority to pull drugs from the market, but the process can take months or even years. Instead, the agency usually makes an informal request and companies almost always comply. Even in the rare cases when drugmakers haven't cooperated, the FDA has prevailed after public hearings and appeals. ___ 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
22-07-2025
- Business
- San Francisco Chronicle
Sarepta shares slide again as drugmaker bows to FDA pressure to pause gene therapy
WASHINGTON (AP) — Shares of drugmaker Sarepta Therapeutics continued to sink Tuesday after the company said it would comply with a Food and Drug Administration request to pause shipments of its gene therapy following several patient deaths. The decision, announced late Monday, comes just days after the company rebuffed FDA regulators in an extremely unusual decision that alarmed investors and analysts. Sarepta CEO Doug Ingram said the company seeks a 'productive and positive' relationship with FDA and that 'maintaining that productive working relationship required this temporary suspension.' The Cambridge, Massachusetts-based company said it would ' temporarily pause all shipments' of its gene therapy Elevidys for muscular dystrophy at the close of business Tuesday. It's the latest in a series of highly irregular moves that have rocked company shares for weeks and forced it to lay off hundreds of staffers. 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. The one-time treatment was initially approved for boys age 4 and younger who could still walk. Last year, FDA expanded approval to older patients who are no longer able to walk. The therapy was already under FDA scrutiny after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. Then the company last week disclosed a third death with a different therapy: a 51-year-old patient who was enrolled in a company trial for another form of muscular dystrophy. FDA responded on Friday by asking the company to immediately halt all shipments of Elevidys. Company executives initially refused, noting that the latest death was not tied to Elevidys, its best-selling product. Wall Street analysts said the company made the right decision to cooperate. Defying the FDA would have 'irreparably damaged the company's relationship with FDA under the current leadership and administration,' TD Cowen analyst Ritu Baral told investors in a note Tuesday. Baral estimated the pause in distribution would last three to six months. The FDA has the authority to pull drugs from the market, but the process can take months or even years. Instead, the agency usually makes an informal request and companies almost always comply. Even in the rare cases when drugmakers haven't cooperated, the FDA has prevailed after public hearings and appeals. ___
Winnipeg Free Press
22-07-2025
- Business
- Winnipeg Free Press
Sarepta shares slide again as drugmaker bows to FDA pressure to pause gene therapy
WASHINGTON (AP) — Shares of drugmaker Sarepta Therapeutics continued to fall Tuesday after the company said it would comply with a Food and Drug Administration request to pause shipments of its gene therapy following several patient deaths. The decision, announced late Monday, comes just days after the company rebuffed FDA regulators in an extremely unusual decision that alarmed investors and analysts. Sarepta CEO Doug Ingram said the company seeks a 'productive and positive' relationship with FDA and that 'maintaining that productive working relationship required this temporary suspension.' The Cambridge, Massachusetts-based company said it would ' temporarily pause all shipments' of its gene therapy Elevidys for muscular dystrophy at the close of business Tuesday. It's the latest in a series of highly irregular moves that have rocked company shares for weeks and forced it to lay off hundreds of staffers. 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. The one-time treatment was initially approved for boys age 4 and younger who could still walk. Last year, FDA expanded approval to older patients who are no longer able to walk. The therapy was already under FDA scrutiny after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. Then the company last week disclosed a third death with a different therapy: a 51-year-old patient who was enrolled in a company trial for a another form of muscular dystrophy. FDA responded by asking the company to immediately halt all shipments of Elevidys. Wall Street analysts said the company made the right move. Defying the FDA would have 'irreparably damaged the company's relationship with FDA under the current leadership and administration,' TD Cowen analyst Ritu Baral told investors in a note Tuesday. Baral estimated the pause in distribution would last three to six months. The FDA has the authority to pull drugs from the market, but the process can take months or even years. Instead, the agency usually makes an informal request and companies almost always comply. Even in the rare cases when drugmakers haven't cooperated, the FDA has prevailed after public hearings and appeals. ___ 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.
Yahoo
11-07-2025
- Business
- Yahoo
TD Cowen Remains Bullish on Amicus Therapeutics (FOLD) With a $20 PT
Amicus Therapeutics, Inc. (NASDAQ:FOLD) is one of the 13 Best Healthcare Stocks to Buy Under $10. On May 1, analyst Ritu Baral from TD Cowen maintained a Buy rating on Amicus Therapeutics, Inc. (NASDAQ:FOLD), keeping the price target at $20.00. Doctors in a lab coat attending to a patient receiving enzyme replacement therapies. The analyst based the rating on the company's future growth potential and strategic positioning, stating that Amicus Therapeutics, Inc. (NASDAQ:FOLD) has maintained a positive outlook for GAAP profitability in H2 2025. This trend emerged despite the Q1 revenue misses for both PomOp and Galafold, which were affected by higher UK VPAG rebates. Baral thus reasoned that these trends suggest confidence in the company's operations and its ability to manage costs and drive future profitability. Another factor supporting the analyst's positive rating is Amicus Therapeutics, Inc.'s (NASDAQ:FOLD) pipeline expansion, especially with the in-licensing of DMX-200 for FSGS. This highlights the company's commitment to boosting its long-term growth prospects and expanding its product offerings, according to Baral. Amicus Therapeutics, Inc. (NASDAQ:FOLD) discovers, develops, and delivers medicines to treat metabolic diseases. Its product portfolio includes the first and only approved oral precision medicine to treat Fabry disease, a clinical-stage treatment paradigm for Pompe disease, and a rare disease gene therapy portfolio. While we acknowledge the potential of FOLD as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 30 Stocks That Should Double in 3 Years and 11 Hidden AI Stocks to Buy Right Now. Disclosure: None. This article is originally published at Insider Monkey.
