Sarepta's Turmoil, FDA Leadership Changes, and Pharma's Manufacturing Investments Shake Industry

Sarepta Therapeutics faces a crisis as the FDA takes decisive action following patient deaths, while the pharmaceutical industry sees significant shifts in regulatory leadership and manufacturing strategies. These developments, coupled with recent drug rejections, signal a changing landscape for drug development and approval processes.

Sarepta's Elevidys Controversy Escalates

Sarepta Therapeutics has encountered severe setbacks following reports of a third patient death related to its gene therapy treatments. The company, which recently announced a major restructuring including 500 layoffs, has now been ordered by the FDA to halt shipments of Elevidys, its Duchenne muscular dystrophy gene therapy. The FDA has also revoked Sarepta's technology platform designation and paused all clinical trials for its limb-girdle muscular dystrophy (LGMD) gene therapy.

The company's stock price plummeted to its lowest point in nearly a decade after media reports revealed a patient death in the LGMD program—information Sarepta had not disclosed during a recent investor call. This lack of transparency has raised concerns about the company's communication practices and the safety of its gene therapy platform.

FDA Leadership Changes Amid Industry Challenges

The FDA's Center for Drug Evaluation and Research (CDER) welcomes a new director, George Tidmarsh, a biotech veteran and adjunct professor at Stanford University's School of Medicine. Tidmarsh assumes leadership at a critical time, as the agency grapples with mass layoffs and voluntary departures.

This leadership transition coincides with a series of high-profile drug rejections. Replimmune and Roche have both received FDA rejections, while therapies from Otsuka/Lundbeck and GSK failed to secure advisory committee support. These outcomes reflect evolving standards for acceptable controls and efficacy demonstrations under FDA Commissioner Marty Makary and CBER Director Vinay Prasad.

Big Pharma Invests Billions in U.S. Manufacturing

In response to potential pharma-specific tariffs and a push for domestic production, major pharmaceutical companies are making substantial investments in U.S. manufacturing. Biogen has pledged $2 billion, while AstraZeneca has committed an impressive $50 billion to enhance their American manufacturing capabilities.

These investments come as President Donald Trump warns of possible tariffs of up to 200% on pharmaceutical imports, which could be implemented as soon as August 1. The industry's proactive approach to reshoring manufacturing operations demonstrates a strategic shift in response to evolving trade policies and a desire to secure supply chains.