FDA sets approval path
for Prosensa muscle disorder drug
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[June 04, 2014]
By Natalie Grover
(Reuters) - 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
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
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 causes DMD.
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
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
[to top of second column]
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.
(This story has been refiled to correct spelling of name in
paragraphs 3 and 4 to De Kerpel from Dekerpel)
($1 = 0.7349 Euros)
(Reporting by Natalie Grover in Bangalore; Editing by Joyjeet Das
and Kirti Pandey)
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