FDA regulator defends decision to OK Sarepta drug

ANDREW HARNIK/ASSOCIATED PRESS/FILE 2015

The nation’s top drug reviewer Wednesday criticized Sarepta Therapeutic Inc.’s program to develop the first drug to treat Duchenne muscular dystrophy, saying the Cambridge biotech’s clinical study involving a dozen patients “left so many questions.”

But in an interview after her appearance at a personalized-medicine conference in Boston, Dr. Janet Woodcock, director of the Food and Drug Administration’s center for drug evaluation and research, said she was nonetheless “very comfortable” with her controversial decision in September to approve the drug for sale. Woodcock said the medicine produced high enough levels of a muscle-building protein to benefit the young boys who are afflicted with the fatal muscle-wasting disease. The results were enough for it to warrant accelerated approval, she said.

“You can have a pretty bad development program, and if you cross the finish line we might approve it anyway,” Woodcock said, who added that Sarepta has been ordered to conduct follow-up studies on patients.

While saying the company’s executives “had a much overblown idea of the pharmacodynamic effect” of their drug candidate, Woodcock brushed aside in-house critics at the FDA and outside advisers who weren’t convinced the drug worked well enough to win approval. They, and others, wondered whether the agency had been unduly swayed by patient advocates who were financially supported by Sarepta.

“I’ve been at FDA for 30 years and we’re always criticized,” Woodcock said. “No matter what decision is made, there’s always somebody unhappy with that decision.”

Feedback from patients is increasingly becoming important to the FDA as it weighs new treatments, she said.

Sarepta executives didn’t respond to a request for comment on Woodcock’s remarks. During her formal presentation at the 12th annual Personalized Medicine Conference at Harvard Medical School, Woodcock didn’t directly address the Sarepta issue. But in an era of more targeted therapies for rare disease, she said regulators must study not only the activity of experimental medicines but the natural history of diseases, including the burden on patients and the rate of progression of symptoms.

“We’ve been talking to patients a lot at the FDA over the last couple of years,” Woodcock said. “They’re really experts in their diseases. They live with it every day. Their perceptions are actually different from the medical models” built by drug developers.

In the case of Duchenne and other deadly rare diseases, patients and their families are demanding faster access to experimental treatments that have the potential to prolong or save lives — even when clinical studies are limited or the results contested.

Woodcock, in the interview, said the FDA is struggling to fill about 700 job openings for drug reviewers and related functions. A big problem, she said, is that agency salaries don’t compare well with what biopharma companies offer the same types of employees. She also faulted the agency’s slow hiring process.

“We need [drug evaluation] people who can go head-to-head with the best scientists in academia and industry,” Woodcock said.

An earlier version of this story incorrectly reported that none of the 12 patients in Sarepta’s clinical trial took a placebo. In fact, four patients took a placebo for the first six months of the trial.