Biopharmaceutical company PTC Therapeutics Inc (NASDAQ:PTCT) reported on Friday the receipt of approval from the US Food and Drug Administration (FDA) for its supplemental New Drug Application (sNDA) for Emflaza to expand its labelling to include Duchenne muscular dystrophy patients between two and five years-old.
Primarily affecting males, Duchenne muscular dystrophy (Duchenne) is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties due to heart and respiratory failure.
In February 2017, Emflaza (deflazacort) was first approved by the US FDA for the treatment of Duchenne in patients five years and older, added the company.
The company said EMFLAZA (deflazacort) is now indicated for the treatment of Duchenne muscular dystrophy in patients two years of age and older.
Under the PTC Cares support programme, the company is dedicated to helping patients, caregivers and prescribers understand the prescription process and financial assistance programnes for PTC products.
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