(Corrects spelling of name in paragraphs 3 and 4 to De Kerpel
* Drug being developed to treat Duchenne muscular dystrophy
* Company to submit U.S. marketing application later this
* Approval expected by 2015 at the earliest - analyst
By Natalie Grover
June 3 Prosensa Holding NV said the U.S.
Food and Drug Administration had outlined an accelerated
regulatory approval path for its most advanced drug, aimed at
treating a muscle disorder.
The Netherlands-based company said on Tuesday it planned to
conduct two more studies, and file for U.S. marketing approval
later this year. Prosensa said it would file for European
approval in the near future.
Like Sarepta Therapeutics Inc's eteplirsen,
Prosensa's drug to treat Duchenne muscular dystrophy (DMD) will
probably win U.S. approval in 2015 at the earliest, leaving each
with about half of the market, said Jan De Kerpel, an analyst at
KBC Securities in Brussels.
De Kerpel expects the drug to generate peak global sales of
1 billion euros ($1.36 billion) by the end of the decade, and
estimates it will cost about 200,000 euros a year per patient.
Prosensa's treatment will likely fare better than Sarepta's
in Europe as it holds a patent advantage in the region, he said.
The company's shares were down about 0.6 percent in
mid-morning trading after rising as much as 3.9 percent.
Drisapersen, like eteplirsen, is designed to enhance the
production of a protein called dystrophin, the lack of which
DMD drugs-in-development have received encouraging signals
from regulators in recent months.
The FDA indicated an alternate path for approval to
Sarepta's eteplirsen in April, while European regulators
recommended conditional approval for PTC Therapeutics Inc's
Translarna last month.
Prosensa said in January it would pursue the development of
drisapersen despite its failure in a late-stage trial, after
additional data showed its use could slow disease's progression.
Earlier that month, partner GlaxoSmithKline Plc
returned the rights to the drug to Prosensa, terminating a 2009
collaboration deal to develop it.
Accelerated approval is usually granted to drugs for serious
diseases with no treatment options, based on data from initial
trials. However, a company still needs to conduct larger trials
to bolster its initial findings.
DMD is a degenerative disorder that hampers muscle movement.
It affects one in 3,600 newborn boys, who usually succumb to the
disease by the age of 30.
Prosensa's shares were trading at $10.56 on the Nasdaq in
mid-morning trading on the Nasdaq.
($1 = 0.7349 Euros)
(Reporting by Natalie Grover in Bangalore; Editing by Joyjeet
Das and Kirti Pandey)