Marathon Pharma submits application for Duchenne muscular dystrophy drug to FDA

pharmafile | June 15, 2016 | News story | Research and Development, Sales and Marketing FDA, duchenne, marathon pharmaceuticals 

Marathon Pharmaceuticals has announced that it has submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for deflazacort, an investigational drug for Duchenne muscular dystrophy.

With this filing, the FDA has a 60-day period to determine whether the NDA is complete and acceptable for filing.

The application is supported by a full data set, including two pivotal clinical efficacy trials for deflazacort in more than 200 Duchenne patients between the ages of five and fifteen. The drug managed to improve muscle strength and other functional outcomes in patients regardless of genetic aetiology and, in one of the studies, ambulation status.

Duchenne muscular dystrophy is an extremely rare genetic disease, affecting one in around 3,600 boys, which cause progressive muscular degeneration. Many sufferers of the disease do not live past the age of 20. There are currently no approved treatments for this disease.

Many pharmaceutical companies have attempted to get their late-stage drugs for this disease approved in the last few months. Biomarin was forced to withdraw its marketing authorisation application for Kyndrisa (drisapersen), following a complete response letter sent by the FDA. Sarepta Therapeutics, meanwhile, is awaiting the FDA’s decision on eteplirsen. Originally criticised by an FDA committee, the regulator’s delay in making a decision has led some to believe it may be granted approval.

Marathon’s deflazacort is not currently approved in the US for any indication. It is available in a number of indications, although not for Duchenne, outside of the US.

Jeff Aronin, chief executive officer at Marathon, says: “This NDA submission starts a process that we hope will result in broad access to this medication for all of those living with Duchenne who need it. We recognise the difficulty the Duchenne community has had in obtaining deflazacort and look forward to working closely with the FDA as they review our application.”

Sean Murray

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