The FDA’s Ronald Farkas was one of Sarepta’s toughest critics. When the biotech made a bid to win an accelerated approval for their Duchenne muscular dystrophy drug, the MD from the division of neurology products shot them down at every turn, spurning the biotech’s shaky case that a tiny trial with just 12 patients had provided any evidence to make it worthy of becoming the first approved drug to treat the lethal, rare disease.
Now, the nemesis of thousands of Sarepta’s advocates in the DMD community has left the agency. A spokesperson confirmed the news, first reported by Benzinga, to Endpoints News on Wednesday morning.
“Dr. Farkas no longer works at the agency,” the spokesperson said in an email statement, declining to amplify on the news of his departure. Soon after, a spokesperson for Parexel also confirmed that Farkas had joined the CRO earlier in the month. He’s now vice president, integrated product development, Parexel Consulting.
And there’s considerable rejoicing among eteplirsen advocates over speculation that his departure may clear a complex hurdle on the way to an approval.
Sarepta’s investors are tapping into that sentiment. The biotech’s shares shot up 26% this morning after rumors of his exit spread overnight. But the argument over eteplirsen’s future is still riding on the outcome of a recent decision to see if the FDA could gather one round of convincing evidence that the drug might work as planned.
Sarepta was able to make its case twice to the FDA. First for a panel meeting that was postponed for weather and a second time in a point-by-point rebuttal of the FDA’s position. None of it worked, though. Farkas and the FDA team didn’t budge, and the panel of experts voted against an approval.
The FDA group has proved equally scathing about other attempts on DMD. PTC was turned away at the door when the agency refused to file its application, citing some major weaknesses. And BioMarin stumbled badly when they rejected their drug, setting the biotech on course to eventually jettison the whole program after the Europeans followed suit.
That left the FDA fending off one of the most aggressive lobbying campaigns it’s ever seen. Patients, families and advocates have pushed hard for an early OK on thin data, insisting that the drug does work. And that push led to the compromise solution to look for some signs that the drug is having the intended effect in spurring the production of dystrophin.
As of now, that’s still where the matter sits, leaving many analysts to wonder just how long the FDA plans to take on this. With Farkas out of the picture, advocates are hopeful that they’ll have an approval soon. But there are no guarantees when it comes to the FDA,
UPDATE: A little after the markets closed Wednesday, Jefferies’ Gena Wang noted that FDA staffers in the neurology division say that Farkas left the FDA a couple of weeks ago. His decision to leave, she adds, was probably made back in August, indicating that the departure had nothing to do with the looming decision on eteplirsen. And he’s been scouting a new position for more than a year.
In a chat with an unidentified key opinion leader in the field, Wang adds that it’s unlikely that Farkas’ departure will make much of a difference in whatever the FDA ultimately decides. The big decision likely rests largely with the director for Office of Drug Evaluation, Dr. Ellis Unger. Also, don’t assume that Farkas’ departure means an approval is coming.
“According to our KOL, a longer delay from the FDA suggests a less likely possibility that the agency has been convinced with newly submitted biomarker data, and additional data could potentially be requested,” Wang adds. “Our KOL also noted, once the PDUFA date has been passed, there is no mechanism to force a decision within a stipulated amount of time.”
It’s worth noting that both sides of this argument have often been wrong about next steps for eteplirsen. This is one story filled with drama and uncertainty.
— Art Krieg (@ArtKrieg) September 13, 2016
The best place to read Endpoints News? In your inbox.
Full-text daily reports for those who discover, develop, and market drugs. Join 17,000+ biopharma pros who read Endpoints News by email every day.Free Subscription