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PTC Therapeutics Receives Complete Response Letter for Ataluren’s NDA

from PTC Therapeutics

SOUTH PLAINFIELD, N.J., Oct. 25, 2017 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Office of Drug Evaluation I of the U.S. Food and Drug Administration (FDA) has issued a complete response letter (CRL) for the New Drug Application (NDA) of the investigational medicine ataluren for the treatment of nonsense mutation dystrophinopathies.

“We are extremely disappointed for the Duchenne community and strongly disagree with the agency’s conclusions,” said Stuart W. Peltz, Ph.D., chief executive officer of PTC Therapeutics. “We believe that this decision fails to consider the benefit-risk of ataluren and the high unmet medical need. Therefore, we plan to file a formal dispute resolution request next week.”

The letter from the Office of Drug Evaluation I of the FDA stated that it is unable to approve the application in its current form. Specifically, the letter indicated that evidence of effectiveness from an additional adequate and well-controlled clinical trial(s) will be necessary at a minimum to provide substantial evidence of effectiveness. The letter also mentioned other nonclinical and CMC matters that PTC is in the process of addressing.

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Eteplirsen Boosts Dystrophin, Function in DMD Details from 12 patients in Study 201/202 reported at AAN

“A detailed look at the trial that won eteplirsen (Exondys 51) FDA approval in Duchenne muscular dystrophy (DMD) showed that the antisense oligonucleotide drug boosted dystrophin production, and improved functional ability compared with historical controls, researchers reported here.”

For the full article published in MedPage Today –  click here.

Capricor Therapeutics Announces Positive Six-Month Results from the Randomized Phase I/II HOPE Clinical Trial in Duchenne Muscular Dystrophy

Capricor Therapeutics, Inc.  a clinical-stage biotechnology company developing first-in-class biological therapies for cardiac and other medical conditions, today announced positive top-line results from a safety and exploratory efficacy analysis of six-month data from the randomized 12-month Phase I/II HOPE Clinical Trial of CAP-1002 (allogeneic cardiosphere-derived cells), an investigational candidate for the treatment of patients with Duchenne muscular dystrophy, or DMD. Duchenne muscular dystrophy is a rare, life-threatening genetic disorder for which treatment options are limited.

Click here for the full release.

CRISPR Corrects Duchenne-Causing Mutations

Using CRISPR-Cpf1 gene editing, researchers have fixed mutations that cause a form of muscular dystrophy in cultured human cardiomyocytes and a mouse model.

To read the article on “The Scientist” – click here