Sarepta Therapeutics is pushing back against the FDA’s request to halt shipments of its muscular dystrophy drug, Elevidys, following concerns about its effectiveness. The company argues that the treatment remains vital for patients, despite mixed clinical trial results. This standoff highlights the tension between regulatory caution and patient access to experimental therapies.

Editor’s Note: This isn’t just a bureaucratic spat—it’s a high-stakes debate over who gets to decide when a drug stays on the market. For families with children suffering from muscular dystrophy, Elevidys represents hope, even if the data isn’t perfect. But the FDA’s job is to ensure treatments actually work, not just that they’re available. The outcome could set a precedent for how regulators and drugmakers navigate these gray areas in the future.

GSK’s stock took a hit after an FDA advisory committee cast doubt on the future of its cancer drug Blenrep, signaling a rocky path ahead for its potential return to the market. While the company insists it still believes in the drug’s safety and effectiveness, investors aren’t so sure—hence the sell-off.

A key FDA advisory panel has rejected GSK's blood cancer drug Blenrep, dealing a blow to the British pharma giant. The drug—aimed at treating relapsed multiple myeloma in patients who’ve already tried other therapies—failed to convince the committee, likely delaying or even halting its U.S. approval path.