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Duchenne Muscular Dystrophy: Business Developments (09.06.13)

September 5, 2013

This is the third Blog Post in a series that examines Duchenne Muscular Dystrophy (DMD) in the rare disease and orphan drug space.

This Blog Post discusses and reviews recent PTC Therapeutics business developments.

PTC Therapeutics, a New Jersey pharmaceutical company, is developing orphan drug Ataluren for DMD.  Ataluren, as described in a recent journal article in Expert Opinion on Orphan Drugs (see References below), is a “stop codon read-through …. gene therapy that allows cells to …. read-through or ignore certain types of mutations called nonsense mutations …. Ataluren introduces a nucleotide sequence at the transfer RNA acceptor site, allowing the translational machinery to continue.” Up to 20% of DMD patients have a premature stop codon.

PTC Therapeutics’ large multinational, randomized, double-blinded, and placebo-controlled, Phase III trial for Ataluren for non-sense-mutation DMD or Becker MD, opens its first site at Cincinnati Children’s Hospital Medical Center, in Cincinnati, Ohio. It is estimated that enrollment is to be completed by mid-2014. There are to be more than 50 trial sites in at least 18 different countries. For full details about this Phase III trial, please reference PTC Therapeutics’ “Frequently Asked Questions (FAQ)” Document.

The company announces on August 26, 2013, the publication of data in the medical journal Muscle and Nerve, that reinforces the 6-Minute Walk Test (6MWT) as a valid primary endpoint for :

  1. Measuring disease progression
  2. Walking ability in ambulatory DMD trials
  3. 20-30 meter range in walking ability is a clinically meaningful change, as measured by the 6MWD.

This analysis is based on natural history data obtained from the company’s Phase IIb trial of Ataluren in 174 nonsense mutation DMD (nmDMD) patients.

The report in Muscle and Nerve represents:

“… an evaluation of the largest multicenter dataset collected to date to determine the reliability and concurrent validity of the 6MWT and other clinical endpoints in DMD. In addition, the research assesses the distribution-based MCID (Minimal Clinically important difference) for 6MWD and other commonly-employed functional endpoints …”.


Review of Phase II and Phase III clinical trials for Duchenne Muscular Dystrophy. Scully M, Pandya S, Moxley R. Expert Opinion on Orphan Drugs. Jan 2013; 1(1): 33-46.

Please Note: “Spinning Device To Mix Tissue Culture Medium” by National Cancer Institute [Public domain] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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