Drugs

PTC Therapeutics NDA Resubmission for Duchenne Drug

FDA has accepted for review a PTC Therapeutics NDA resubmission for Translarna (ataluren) and its use in treating nonsense mutation Duchenne muscular dystrophy. In 2017, the agency issued a complete response letter on the original NDA, which called for effectiveness evidence from an additional adequate and well-controlled clinical trial. The letter also mentioned other nonclinical and manufacturing matters that PTC said it would address.

The company says the resubmission is based on data from a placebo-controlled trial (Study 041). “Following 72 weeks of Translarna treatment, there was significant benefit demonstrated on the key study endpoints of six-minute walk distance (6MWD) (p=0.0248), NorthStar Ambulatory Assessment (p=0.0283), 10-meter walk/run (p=0.0422), four-stair climb (p=0.0293), and time to 10% worsening of 6MWD (p=0.0078),” the company says.

Additionally, PTC says Therapeutics the filing includes the findings on long-term Translarna treatment benefit as documented in the STRIDE registry. “Translarna treatment resulted in a 3.5-year delay in loss of ambulation (p<0.0001) and a 1.8-year delay in reaching a predicted forced vital capacity of less than 60% (p=0.0028) a critical threshold of lung function,” it adds.

Translarna is described as a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation, which is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. “The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne,” the company says.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome