PTC Initiates Rolling NDA for Nonsense Mutation DMD Treatment Translarna

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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PTC TherapeuticsSouth Plainfield, NJ-based biopharmaceutical company PTC Therapeutics, Inc. recently announced that it has launched a rolling submission of a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for pipeline nonsense mutation Duchenne muscular dystrophy (nmDMD) treatment Translarna™. This process enables the completed portions of the application to be submitted and reviewed by the agency on an ongoing basis. The company foresees a finalization of the NDA by Q4 2015, after the completion of a confirmatory Phase III ACT DMD clinical study.

Translarna is a novel protein restoration therapy, formulated to encourage production of functional protein in patients with nonsense mutations, such as the non-expression of dystrophin in DMD. At present, Translarna is approved in the European Economic Area as a treatment for nmDMD in ambulatory patients aged 5 years and older. In the United States, the drug’s development is funded by the Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation); Muscular Dystrophy Association; FDA’s Office of Orphan Products Development; National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy.

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“The initiation of our NDA submission for Translarna marks another significant milestone towards providing Translarna to all nonsense mutation Duchenne muscular dystrophy patients,” said Stuart W. Peltz, Ph.D., the Chief Executive Officer of PTC Therapeutics, Inc. “We look forward to the completion of the ACT DMD confirmatory Phase 3 clinical trial so that we can finalize the NDA. Gaining US approval, in addition to Translarna’s European approval, will help to make Translarna available to patients across the globe. This is our commitment to the patients, families, advocacy groups and physicians who have worked and supported PTC Therapeutics through many years of research and development.”

Pat Furlong, the president and founder of Parent Project Muscular Dystrophy, a not-for-profit organization founded in 1994 by parents of children with Duchenne and Becker Muscular Dystrophy, said there is a large unmet clinical need for a treatment like Translarna in the United States. The muscular dystrophy community lauds PTC Therapeutics’ hard work and determination in expediting the drug’s US availability.

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