Sept 14 Shares of Sarepta Therapeutics Inc
rose as much as 29 percent after a key critic of its
muscle-wasting drug left the U.S. Food and Drug Administration
ahead of the agency's decision whether to approve it.
Sarepta's duchenne muscular dystrophy (DMD) drug,
eteplirsen, has been in the spotlight this year with patient
groups and parents arguing passionately in favor of the
treatment to pressure the regulator to approve it.
The FDA confirmed on Wednesday that Dr. Ronald Farkas no
longer works at the agency. The regulator did not provide any
details about Farkas's departure.
Farkas was seen as the leading voice within the FDA arguing
against the approval of the drug and the author of a scathing
critique of the company's application.
Farkas's review, which was a part of briefing documents
published ahead of an April advisory committee meeting, said
there was no clear evidence the drug slowed progression of the
Oppenheimer analysts said Farkas's departure may signal that
eteplirsen decision is imminent and that they continue to
believe that the drug will be approved.
Dr. Janet Woodcock, the head of the FDA's pharmaceuticals
division, appeared willing from the beginning to support
approval pending the outcome of a confirmatory trial.
Suntrust Robinson Humphrey said Farkas's departure at this
juncture would further indicate that there is turmoil within the
FDA on what to do with eteplirsen.
The brokerage said from an empirical standpoint, the only
decision likely to come from the FDA should be a denial of
"But with emotions and advocacy groups and likely political
pressure coming to bear, it could turn what we believe should be
a clearly derived outcome into another irrational 180-degree
turn with the agency."
The FDA in June asked Sarepta for additional data after the
agency delayed its highly anticipated decision on whether to
approve the drug.
The advisory committee meeting, where patients hurled angry
comments at Farkas, was the latest example of the growing impact
of patient lobbying in influencing the FDA's decision-making.
The agency has formally committed to increasing the role of
patients, but patient advocates are increasingly packing
advisory committee meetings in the hope of influencing the
panelists, even in cases where data presented by the companies
Duchenne muscular dystrophy is a rare genetic disorder
characterized by progressive muscular weakness and is caused by
a lack of dystrophin, a protein needed to keep muscles healthy.
Eteplirsen is designed to increase the production of dystrophin.
Sarepta shares were up about 26 percent at $32.20 on
Wednesday. Up to Tuesday's close, the stock had fallen 33.6
percent this year.
(Reporting by Ankur Banerjee in Bengaluru; Additional reporting
by Toni Clarke in Washington; Editing by Saumyadeb Chakrabarty)