The Committee for Medicinal Products for Human Use (CHMP) of the
European Medicines Agency (EMA) provided a positive opinion on the
marketing authorization of
PTC Therapeutics, Inc.
) Translarna. Shares of PTC Therapeutics shot up around 30.7%
following the news.
PTC Therapeutics is looking for a conditional approval for
Translarna for the treatment of nonsense mutation Duchenne muscular
dystrophy (nmDMD) in ambulatory patients of five years and above.
We note that the conditional marketing authorization will allow the
company to market the product with unified labelling in the 28
countries that are Member States of the EU, as well as European
Economic Area members Iceland, Liechtenstein and Norway.
A final decision from the European Commission on the approval of
Translarna is expected by Aug 2014. The positive opinion from the
CHMP is a huge relief for the company. In Jan 2014, the EMA
advisory body had provided a negative opinion on the approval of
Translarna. The company had then requested the CHMP for a
re-examination of the opinion.
We note that the favorable opinion from the CHMP was based on
positive data from a phase IIb, double-blind, placebo controlled
study on Translarna. Results from the study revealed that patients
with nmDMD under Translarna treatment walked an average of 31.3
meters more than patients under placebo at week 48. Translarna
treated patients demonstrated a slower rate of decline in
ambulation as well. Translarna was also well tolerated during the
PTC Therapeutics is currently running a confirmatory, phase III,
ACT DMD study on Translarna. The company intends to complete
enrollment for the study by mid 2014. PTC Therapeutics believes
that results from the study will help them to gain full EU approval
of Translarna. The company also intends to file for marketing
approval application in the U.S. on the basis of the results from
the ACT DMD study.
We are encouraged by the positive opinion from the CHMP on the
approval of Translarna. We expect investors to stay focused on the
company's progress with Translarna going forward. The successful
development of Translarna is crucial for the company.
PTC Therapeutics, a biopharma company, presently carries a Zacks
Rank #3 (Hold). Some better-ranked stocks in the same sector
Gilead Sciences Inc
Alexion Pharmaceuticals, Inc.
AVEO Pharmaceuticals, Inc.
). While Gilead carries a Zacks Rank #1 (Strong Buy), Alexion and
AVEO hold a Zacks Rank #2 (Buy).
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