FDA accepts Marathon’s Duchenne drug for priority review
pharmafile | August 11, 2016 | News story | Research and Development, Sales and Marketing | Duchenne Muscular Dystrophy, FDA, deflazacort, marathon, marathon pharmaceuticals
Marathon Pharmaceuticals has announced that the US Food and Drug Administration (FDA) has accepted and granted a priority review for deflazacort, its investigational drug for the rare genetic disease, Duchenne muscular dystrophy.
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. Using trial data to prove efficacy is difficult, given the small treatment population.
Several companies have put forward drug candidates for Duchenne in recent months, which resulted in delays and job cuts. Marathon announced its filing for a new drug application for deflazacort in June.
In a statement, Marathon indicated that its filing included trial data which showed improvements in muscle strength and other functional outcomes in Duchenne patients receiving deflazacort.
Jeff Aronin, CEO at Marathon, says: “This is an important milestone for Maraton but more importantly, for the Duchenne community. Today, a large number of those living with Duchenne who could potentially benefit from deflazacort do not get it simply because they do not have access. If deflazacort is approved, our goal is to work closely with the community and to make deflazacort widely available to Duchenne patients in the US.”
Valerie Cwik, chief medical and scientific officer for the Muscular Dystrophy Association, comments: “The Duchenne community would greatly benefit from widespread and reliable access to a treatment option with the potential to delay disease progression. We are all too familiar with the challenges that children and adults with Duchenne and their families face, and we’re hopeful that an FDA approval of deflazacort would be one of the first of many treatments for Duchenne.”
Sean Murray
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