PTC Therapeutics has announced it will submit the results its Phase 3 ACT DMD clinical trial for review by Health Canada as part of the New Drug Submission (NDS) for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
DMD is a progressive disease that mostly affects male patients and is caused by a lack of functional dystrophin protein, which is critical to the structural stability of skeletal, diaphragm, and heart musculature. Patients who suffer from the most severe form of this condition eventually stop walking as early as age 10 and experience life-shortening lung and heart complications in their late teens and early 20s. The medical community estimates that nmDMD affects nearly 13 percent of all DMD patients.
According to a press release, to submit this supplementary data for review, PTC will withdraw its current NDS from Health Canada and then resubmit it with the added ACT DMD results. PTC no longer expects Health Canada to review its NDS application in the first half of 2016.
PTC’s proprietary small molecule drug targets a specific mechanism of RNA biology, commonly referred to as post-transcriptional control, which includes the regulatory events that occur in cells during and after a messenger RNA, or mRNA, molecule is being copied from DNA.
Translarna (ataluren), the protein restoration drug candidate being advanced by PTC Therapeutics, is designed to enable the formation of a new, functional protein in patients with genetic conditions caused by a nonsense mutation – a modification in the genetic code that stops the synthesis of an essential protein. The disorders that may result from this mutation are determined by which proteins are being kept from becoming fully functional, including DMD.
Translarna is licensed in the European Economic Area (EEA) for the treatment of nmDMD in ambulatory patients who are at least 5 years old. In the United States and Canada, Translarna is an Investigational New Drug (IND) with its development grounded on grant support from organizations such as the Cystic Fibrosis Foundation Therapeutics Inc. , the nonprofit affiliate of the CF Foundation; Parent Project Muscular Dystrophy (PPMD); National Heart, Lung, and Blood Institute (NHLBI); National Center for Research Resources (NCRR); the FDA’s Office of Orphan Products Development (OOPD); and the Muscular Dystrophy Association (MDA).