Turmoil at the FDA means uncertainty for innovative new drugs

The Food & Drug Administration has long been considered the gold standard among federal regulatory agencies, putting experimental drugs through rigorous and impartial testing and scrutiny to ensure their safety and efficacy before granting market approval.

But in the last few days the FDA’s oversight has left the biopharma industry confused at best as it reversed itself twice over one controversial drug, Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy. Then on Tuesday night Dr. Vinay Prasad, the FDA official overseeing those reversals, resigned unexpectedly after less than four months on the job and considerable criticism from conservative commentators.

In a commentary in Endpoints News, a closely followed drug industry newsletter, executive editor Drew Armstrong wrote that “It’s hard to read that series of events as anything other than the FDA caving to outside politics.”  

Wall Street analysts are divided on whether Prasad’s departure will be positive for the biotech industry. Prior to his appointment as director of the FDA’s Center for Biologics Evaluation and Research (CBER), which oversees the review of vaccines and gene and cell therapies, Prasad was a long-time critic of the FDA’s policies and drug approvals, gaining considerable attention for this criticism of Covid-19 vaccines.

BMO Capital Markets analyst Evan Seigerman wrote in a note that Prasad’s departure is a “major positive.” for the biotech industry. “While we often see uncertainty as a negative for the sector, this change of Prasad at CBER may actually instill some confidence and stability in FDA.”  

But Jefferies analyst Andrew Tsai wrote that “We felt Dr. Prasad thought more rationally (and conservatively) around FDA approvals, requiring sponsors to produce more supporting clinical evidence.” He added that “events have seemed relatively more predictable under Prasad, as there was a sense of structure, framework, and merit behind CBER’s decisions.”

Stifel analysts, meanwhile, said in a note that Prasad’s departure “might eventually be good but how can we know?”

Prasad’s departure was prompted by a controversy centered on the Sarepta drug Elevidys, which won accelerated approval from the FDA in 2023 as the first gene therapy treatment for 4- and 5-year-old children with Duchenne muscular dystrophy who could still walk. The muscle wasting disease, caused by a defective gene, affects one in every 3,300 boys; there is no known cure and few treatments.

Elevidys, administered by a single infusion, delivers a corrective gene that helps prevent the degeneration of muscle cells. It is one of the world’s most expensive drugs, costing $3.2 million for the one-time treatment.

In 2024 the FDA expanded Elevidys’s approval to include ambulatory and non-ambulatory Duchenne patients who were 4 years of age or older. Both approvals were made by Dr. Peter Marks, Prasad’s predecessor, who resigned under pressure in March. The approvals were controversial as the clinical trial data for Elevidys was not conclusive that the drug could significantly improve motor function, but there was considerable pressure on the agency from families of Duchenne patients to move ahead on the drug, since they have so few options.

One of the most vocal critics of the Elevidys approval was Prasad himself, who directly criticized Marks’ decision to approve Sarepta’s Duchenne muscular dystrophy drug, overruling recommendations by FDA advisors that the drug be rejected.

Consequently, Prasad was quick to call on Sarepta to temporarily pull the drug from the market earlier this month after two teenagers taking Elevidys died from acute liver failure, and an adult muscle dystrophy patient on a different but related drug died.  Sarepta initially refused, an unusual confrontation with the agency, but then agreed on July 21 to shelve the drug.

Sarepta’s stock plunged on the news, and speculation was rife that Elevidys would not return, since it would likely need to go through another round of clinical trials.

Prasad’s critics quickly went into action. On Monday, July 28, the Wall Street Journal’s lead editorial, headlined The Sarepta Mugging and Drug Innovation, called the FDA’s action “a regulatory mugging,” and referred to Prasad as “a self-professed Bernie Sanders acolyte who dislikes the drug.”

The Journal editorial board went on to say that “Dr. Marks made good decisions to prioritize speed and patient choice over certainty on the data” and concluded that “families facing a terrible diagnosis are left wondering if the only gene therapy on the market will be an option for their sons. If Americans wanted fewer novel treatments, they could have elected Kamala Harris.”

Conservative activist Laura Loomer, a favorite of President Trump, was another Prasad critic, writing recently that “Prasad’s regulatory philosophy is fundamentally anti-Trump. While the President has criticized the FDA’s ‘slow and burdensome’ approval process, he has argued for tougher reviews. Also, Prasad dismissed Republican pressure to streamline approvals as misguided.” And on X she posted, “How did this Trump-hating Bernie Bro get into the Trump admin???”

Trump previously fired several national security officials after Loomer accused them of being insufficiently loyal to the president.

By Monday evening, the FDA told Sarepta that it would lift its pause on Elevidys, Late Tuesday, Prasad resigned. “Dr. Prasad did not want to be a distraction to the great work of the FDA in the Trump administration and has decided to return to California and spend more time with his family,” the Department of Health and Human Services, which oversees the FDA, said in a statement. “We thank him for his service and the many important reforms he was able to achieve in his time at FDA.”

HHS has not named a successor to Prasad. His departure comes during a period of historically high turnover among the leadership of the FDA, including the loss of Jacqueline Corrigan-Curay, the top drug regulator at the agency, who announced her retirement last month.

“With Dr. Prasad’s time as director of CBER now over, we wonder if his departure could signal a shift towards the more permissive, patient advocacy centered ‘right to try’ wing of the MAHA [Make America Healthy Again] movement vis-à-vis rare disease indications,” wrote Leerink Partners in reaction to his resignation.

As for Sarepta, its shares were up 15.4% on Wednesday, with Prasad’s departure widely seen as a victory for the company.