FDA's New Drug Boss to Retire in January Ahead of Trump Reform Efforts

December 6, 2016
By Mark Terry, BioSpace.com Breaking News Staff

Ahead of President-Elect Donald Trump being sworn in on January 20 with promises to reform the U.S. Food and Drug Administration (FDA), John Jenkins, longtime director of the FDA’s Office of New Drugs will retire.

What makes this particularly notable is Jenkins’ recent criticisms of Sarepta Therapeutics ’ Exondys 51 (eteplilrsen) for Duchenne Muscular Dystrophy (DMD), and recent internal battles over the drug between Jenkins and others with Janet Woodcock, Director of the FDA’s Center for Drug Evaluation and Research (CDER). Woodcock will be taking over for Jenkins until a replacement is found.

There was significant internal and external debate of Exondys 51. Many FDA scientists felt that Sarepta had not proven the drug’s efficacy. DMD is a muscle wasting disease caused by mutations in the dystrophin gene. It is a progressive disease that usually causes death in early adulthood, with serious complications that include heart or respiratory-related problems. It mostly affects boys, about 1 in every 3,500 or 5,000 male children.

Dissent inside the FDA over the drug was led by Ronald Farkas, clinical team leader, who left the agency just prior to approval. The agency’s acting chief scientist, Luciana Borio, and Ellis Unger, director of the office of drug evaluation, also strongly opposed approval, arguing that Sarepta did not provide substantial evidence of Exondys 51’s effectiveness.

But Woodcock pushed it through, overruling her staff. The final decision went to Robert Califf, the FDA’s Commissioner. Internal reports suggest that Califf had similar reservations as Borio and Unger, but sided with Woodcock.

On October 18, Jenkins, gave a talk at the NORD Summit Meeting that directly focused on the decision. He specifically stated, “The path taken by Sarepta is NOT a good model for other development programs.”

Woodcock has long argued that the agency needs to be more flexible in how it approaches drug approval, especially when it comes “to a devastating illness with no treatment options.”

The argument appears to be along the lines of: The company hasn’t proved that it works, but it’s the only one we’ve got, so we’ll approve it.

In his talk, Jenkins said, “Flexibility in FDA regulations does not mean marketing approval prior to demonstration of substantial evidence of effectiveness. NOT as a ‘rescue’ for a failed program.”

John Carroll, writing for Endpoints News, says, “But no matter how people sided in the civil war that divided regulators on Sarepta, there’s no doubt that Woodcock’s already considerable clout at the FDA just grew by several degrees, leaving her in a position to reform the OND on her own terms as the FDA preps for the arrival of Donald Trump at the White House.”

The concern of critics is that with additional, although probably short-term clout at the FDA, Woodcock will mold the OND more along the lines of her thinking in terms of “flexibility.” However, that is also likely to depend a great deal on who President-Elect Trump chooses to be the next FDA Commissioner, as well as who ends up running the OND.

Michael Gaba, the federal policy leader of law firm Holland & Knight’s life sciences team, told Carroll, “I expect that the opposition expressed by Jenkins and others within the FDA leadership around the Sarepta approval will continue among drug reviewers as they are bound to ensure products are safe and effective for their intended use as supported by scientific data.” But, he goes on to say, there are concerns if more and more companies try to push things through the way Sarepta did. “This could, over time I suspect, tarnish the FDA’s ‘good housekeeping seal of approval’ that industry as a whole should want to uphold.”

Jenkins spent almost 25 years at the FDA and will retire on January 6, 2017. He has led the OND for the last 15 years, overseeing a staff of more than 1,000, while also interacting with the biopharma industry. Woodcock wrote an email to staff making the announcement, saying, “With John at the helm, OND has effectively used various regulatory and scientific tools to ensure safe and efficient development, review, and approval of new therapies—all while maintaining high standards for safety, effectiveness, and quality.”