(Adds detail from CHMP statement, analyst comments; updates
By Vrinda Manocha
Jan 24 PTC Therapeutics Inc said its
drug to treat a rare muscular disorder failed to win support for
a conditional approval from a panel of the European Medicines
Agency, sending its shares down as much as 17 percent in morning
The European Committee for Medicinal Products for Human Use
said a study failed to show that patients taking the drug,
ataluren, could walk a greater distance in six minutes than
patients on placebo. (link.reuters.com/vew36v)
It also said the drug showed limited evidence of having
beneficial effects on the daily activities of patients with a
form of Duchenne muscular dystrophy (DMD), a genetic disorder
that causes muscle degeneration and eventual death in boys.
Analysts said conditional approval for the drug had been
considered unlikely, and remained positive on its potential for
eventual approval by the EMA.
A conditional approval is granted to treatments that address
unmet medical needs. The drugmaker is still testing the drug in
a late-stage trial.
The EMA was concerned that conditional approval would make
it difficult for the company to finish the trial, Wedbush
analyst Christopher Marai said.
PTC said it plans to request the committee for a
re-examination, but analysts said they expect the company would
need data from its late-stage trial of the drug before it is
Marai said the drug could be approved by late 2015 or early
2016 and would target about 1,500 patients in the European
Union. "The drug could generate peak sales of $350 million by
2019 in the European Union," he said.
PTC joined a list of companies struggling with setbacks to
their drugs to treat DMD.
Prosensa Holding NV's partner GlaxoSmithKline Plc
handed back the rights to its DMD drug less than two
weeks ago after the failure of a key trial.
Sarepta Therapeutics Inc lost almost two-thirds of
its market value in November after the U.S. FDA said the design
and goals of its trial might not be sufficient to get approval
for its muscular disorder drug.
About 15 percent of DMD cases are caused by nonsense
mutations that leads to a premature end to the translation of a
gene into a protein, according to the U.S. National Institutes
Ataluren is designed to interact with a part of the cell
known as a ribosome, enabling it to manufacture a protein called
dystrophin, the absence of which causes muscle weakening in
patients with DMD.
The company's shares were trading down 4 percent at $24.20
in morning trading on the Nasdaq.
(Reporting by Vrinda Manocha in Bangalore; Editing by Don