PTC Therapeutics buys rights to DMD drug Emflaza

16 March 2017

PTC Therapeutics (Nasdaq: PTCT) saw its shares decline 11.7% to $9.64, after it revealed that it has entered into an asset purchase agreement with privately-held Marathon Pharmaceuticals to acquire all rights to Emflaza (deflazacort).

In February this year, Emflaza became the first treatment approved in the USA for all Duchenne muscular dystrophy (DMD) patients five years and older, regardless of their genetic mutation. Emflaza aligns with PTC's mission to bring therapies to patients who have rare diseases with limited or no treatment options, stated the company, whose own DMD drug Translarna (ataluren) was given conditional approval in Europe in 2014, but is still awaiting clearance in the USA.

Hefty price tag previously criticized

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