PTC Therapeutics to file new Phase III data for genetic muscle disorder drug in Canada

pharmafile | March 15, 2016 | News story | Manufacturing and Production, Research and Development Canada, PTC Therapeutics, Translarna, nonsense mutation Duchenne muscular dystrophy, phase III 

PTC Therapeutics (Nasdaq: PTCT) said it does not expect the Canadian regulators to review its trial drug to treat a genetic muscle-wasting condition in the first half of 2016 as it plans to submit results from a new trial.

The company said it plans a New Drug Submission (NDS) following the results of the late-stage trials for its Translarna (ataluren) to treat nonsense mutation Duchenne muscular dystrophy (nmDMD).

Translarna is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.

A nonsense mutation 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 muscular dystrophy.

Translarna is licensed in the European area to treat nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older.

Duchenne muscular dystrophy is a progressive muscle disorder caused by the lack of functional dystrophin protein and primarily affects males.

Anjali Shukla

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