|
 Shares of the company plunged 12.7% to $105.05 in trading after the
bell.
Analysts had expected annual sales of nearly $400 million in five
years for golodirsen, the second treatment from Sarepta for DMD, a
rare muscle-wasting disorder that mainly affects boys.
The Cambridge, Massachusetts-based company is also in a close race
with Pfizer Inc and Solid Biosciences Inc to introduce a gene
therapy for DMD in the coming years.
Sarepta acknowledged that kidney toxicity was observed in animal
trials, which tested the drug, golodirsen.
However, the doses in the animal trials were "ten-fold higher" than
those tested on humans, and the side effect was not observed in the
human trial on which the application for approval was based, the
company said.
"We are very surprised to have received the complete response letter
this afternoon," Chief Executive Officer Doug Ingram said in a
statement.
"Over the entire course of its review, the agency did not raise any
issues suggesting the non-approvability of golodirsen, including the
issues that formed the basis of the complete response letter,"
Ingram said.
The FDA, in a statement, said it sees the unmet medical need in DMD
and the urgency to make new treatments available.
"We will continue to work with companies and the patient community
to facilitate development and approval of safe and effective
treatments for DMD," the health agency said.
Duchenne muscular dystrophy is a progressive disorder that begins to
cause symptoms at an early age, first by hampering the ability to
walk and later by causing breathing difficulties and heart problems.
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Many patients die at a relatively young age from DMD, which is
caused by the absence of dystrophin, a muscle building protein.
Sarepta's golodirsen skips a faulty section of the related gene, in
this case called 'exon 53', to produce dystrophin.
Exondys 51, the company's first DMD treatment, was approved in 2016
against the advice of the FDA's outside panel of experts and its own
reviewers, who had questioned the drug's effectiveness.
Exondys' average net price based on a patient's weight is $300,000 a
year, the company said.
Sarepta had planned to price golodirsen at the same price as Exondys
51, Chief Executive Officer Douglas Ingram said in an interview
prior to the FDA response.
Exondys, which targets a different mutation that affects about 13%
of DMD patients, brought in sales of $301 million in 2018 and
analysts expect sales to grow to $465.56 million in 2024, according
to Refinitiv data.
Marathon Pharmaceuticals' drug, Emflaza, a steroid-based therapy,
was approved to treat the disorder in 2017. PTC Therapeutics Inc,
which sells another DMD treatment in Europe and certain other
geographies, later bought the U.S. rights to Emflaza.
Also coming are gene therapies being developed by Sarepta and Pfizer
Inc, which aim to help patients keep producing dystrophin after a
one-time infusion.
Sarepta said it would request a meeting with the FDA to determine
next steps for golodirsen.
(Reporting by Manas Mishra and Nivedita Balu in Bengaluru; Editing
by Anil D'Silva, Bernard Orr)
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