CHMP Recommends Renewal of Translarna’s™ Marketing Authorization for Nonsense Mutation Duchenne Muscular Dystrophy Based on Continued Positive Benefit-Risk Assessment

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients five years and older. In connection with the renewal, the marketing authorization will include a specific obligation to conduct an additional long-term post-authorization trial.

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Capricor Therapeutics Announces Plans to Expand Clinical Development Program in Duchenne Muscular Dystrophy to Evaluate Peripheral and Respiratory Muscle Function

Capricor Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology company developing first-in-class biological therapies for cardiac and other serious medical conditions, today announced that it intends to expand its CAP-1002 clinical development program in Duchenne muscular dystrophy (DMD) to encompass the skeletal muscle aspects of the disease, in addition to the cardiac complications.

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RASRx

Approach: Anti-fibrotic
Funding Impact: Accelerated identification of lead compound and de-risked pre-clinical program

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Questions Regarding Deflazacort Access and Regulatory Status

This informational webinar highlighted the regulatory process to date, updated the community on Marathon Pharmaceutical’s Expanded Access Program (EAP) for deflazacort and discussed what a potential FDA approval of deflazacort would mean in terms of access to the drug in the United States. Some of those who import versions of deflazacort from abroad may have received an update from their distributor with initial details, which was discussed along with steps deflazacort patients might choose to take to help ensure continued access to U.S. supply of deflazacort if FDA approval is granted.

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MyoTherix

Approach: Anti-inflammatory, anti-osteoporosis, potential steroid sparing therapies
Funding Impact: Industry partnership to sponsor clinical trial for Duchenne

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Better Designed Clinical Trials Now

  As a parent of a son with Duchenne I have been frustrated that the data presented to the FDA does not match the patient experience.  I personally know so […]

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