The FDA's lead reviewer, Dr. Ronald Farkas, recently quit the agency
after issuing a scathing report criticizing the quality of the data
presented by Sarepta Therapeutics Inc, developer of the drug Exondys
51, known also as eteplirsen.
Sarepta's stock nearly doubled to $56.18 on news of the decision.
Farkas's departure highlighted sharp divisions within the agency. He
and other scientists were opposed by Dr. Janet Woodcock, the
agency's powerful head of pharmaceuticals, who argued for approval,
according to a summary of the dispute.
In an email to staff on Monday, Woodcock said the approval "reflects
FDA's ability to apply flexibility to address challenges we often
see with rare, life-threatening diseases - while remaining within
our statutory framework."
The drug treats a subset of patients with Duchenne muscular
dystrophy, a rare, progressive genetic disorder that hampers muscle
movement, eventually killing most sufferers by age 30. The subset
includes about 13 percent of all DMD patients, or some 1,300 to
1,900 patients in the United States.
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FDA scientists, including Dr. Ellis Unger, director of the drug
evaluations division overseeing the product, appealed Woodcock's
planned decision to an internal disputes board, according to a
publicly available summary of the dispute written by FDA
Commissioner Robert Califf on Sept. 16.
The agency's acting chief scientist, Dr. Luciana Borio, also did not
believe Sarepta's data supported approval, Califf's summary stated.
Nonetheless, Califf decided to "defer to Dr. Woodcock's judgment and
authority to make the decision."
"I find no basis for a view that Dr. Woodcock was unduly influenced
by involvement with the patient community or other external
pressures," he wrote.
He added, however, that "serious shortcomings present in the
eteplirsen development program should not be allowed to establish a
broad precedent for therapeutic development in rare diseases."
Simos Simeonidis, an analyst at RBC Capital Markets, described the
approval as "one of the most perplexing regulatory decisions in
recent history."
The FDA approved Sarepta's drug under its so-called "accelerated"
approval pathway in which a product is approved based on data
believed to predict a clinical benefit. That benefit must be proven
by the company in a subsequent clinical trial.
"It will be years before we find out the outcome of that trial,"
Simeonidis said, adding that Sarepta "now becomes one of the most
attractive mergers and acquisitions targets in biopharma."
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PATIENT ADVOCATE PRESSURE
This is the second time in just over a year that the FDA has bowed
to patient pressure to approve a drug despite scant scientific
evidence showing it worked.
Last August the agency approved Addyi, a pill to boost libido in
women with low sexual desire. A coalition of women's groups backed
by the manufacturer, Sprout Pharmaceuticals, packed an advisory
committee meeting with women who testified to their desperate need
for the pill.
Woodcock said at the time that the agency was "committed to
supporting the development of safe and effective treatments for
female sexual dysfunction." The drug carries a boxed warning saying
it can cause fainting and extremely low blood pressure and that it
should not be used with alcohol.
Analysts said the approval of Exondys 51 bodes well for similar
approvals elsewhere in the world. Tim Lugo, an analyst with William
Blair, estimated the drug will generate global peak annual sales of
close to $2 billion.
The FDA also granted Sarepta a rare pediatric disease voucher
representing a commitment by the FDA to review a new drug developed
by Sarepta within six months rather than the standard 10 months or
more.
The voucher can be sold to another company. Michelle Gilson, an
analyst at Oppenheimer, estimates the voucher could be worth roughly
$350 million, which could be used to fund the launch of the drug.
BioMarin Pharmaceutical Inc's Kyndrisa, designed to address the same
subset of patients as eteplirsen, was rejected by the FDA in
January.
(Editing by Andrew Hay and David Gregorio)
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