* Plans to file for eteplirsen approval by end of 2014
* Stock soars as much as 64 pct
* Rival Prosensa's shares jump as much as 12 pct
(Recasts throughout; adds background, analyst comment,
conference call details)
By Esha Dey and Natalie Grover
April 21 Sarepta Therapeutics Inc's
shares soared 64 percent after it said that the U.S. Food and
Drug Administration indicated an alternate path to approval for
the company's experimental muscle disorder drug.
The news provides a fresh lease of life for Sarepta, whose
plans for a marketing approval the regulator deemed "premature"
late last year, citing insufficient trial data.
The agency has now indicated that safety and efficacy data
from studies without placebo groups could support an approval
application, analysts said.
The regulator had earlier said that the company would need
to compare the drug, eteplirsen, with a placebo.
Patients treated with the drug in the new trials can now be
compared with historical data from other patients with Duchenne
muscular dystrophy (DMD), analysts said.
Sarepta plans to file for approval by the end of the year
and expects to get marketing nod in the second half of 2015.
"Currently we have 2018 as the year of launch for
eteplirsen, but this timeline could shave as much as 2+ years
off that projection," Cowen & Co analyst Edward Nash said in a
Sarepta is developing eteplirsen as a treatment for DMD, a
degenerative disorder that hampers muscle movement and affects
one in 3,600 newborn boys - who usually succumb to the disease
The biotechnology company said it would submit its marketing
application for eteplirsen under an accelerated approval
pathway, as suggested by the FDA.
Accelerated approval is usually granted to drugs for serious
diseases with no treatment options, based on initial trial data.
However, the drugmaker still needs to conduct larger trials to
bolster its initial findings.
Monday's news lifted the shares of Prosensa Holding NV
, which is developing a rival DMD drug, drisapersen.
Prosensa's drug had failed to improve walking ability in DMD
patients in a late-stage trial in September 2012.
Drisapersen, like eteplirsen, is intended to work by
enhancing the production of a protein called dystrophin, the
lack of which causes the disease.
Other attempts to develop a treatment for DMD have also been
PTC Therapeutics Inc failed to win support for a
conditional approval of its treatment from a panel of the
European Medicines Agency in January.
Cambridge, Massachusetts-based Sarepta's shares plummeted 64
percent to $13.16 on Nov. 12 last year when the FDA raised
doubts on the drug's trial data, but have now regained most of
The stock was up about 49 percent at $36.37 in afternoon
trade on the Nasdaq on Monday.
(Reporting by Esha Dey and Natalie Grover in Bangalore; Editing
by Sriraj Kalluvila and Savio D'Souza)