Emflaza (deflazacort) was approved by the FDA in February 2017 to treat children over the age of five with Duchenne muscular dystrophy (DMD). Unfortunately, due to issues with pricing, the drug hasn’t been available to the public.
The drug, which was originally developed by Marathon Pharmaceuticals, has since been acquired by PTC Therapeutics. The acquisition has renewed hope in the MD community that the drug will soon become available, and at a much lower price than the $89,000 originally quoted by Marathon. Find out more about this story here.
Emflaza is a corticosteroid that reduces inflammation and decreases immune system reaction. Clinical trials of the drug found that it improved muscle strength in boys with Duchenne MD and reduced the time taken to perform function tests like walking 30 feet and climbing four stairs.
Deflazacort has been used by Duchenne patients in other countries for some time, but this is the first time the FDA has approved its use for any medical reason.
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