No slam dunk, but DMD data could secure future for Translarna

22 June 2022

After repeated failures to register its Duchenne muscular dystrophy (DMD) drug in the USA, new data suggest PTC Therapeutics (Nasdaq: PTCT) might be about ready to try again.

The New Jersey, USA-based rare disease drug developer has long had conditional marketing approval in Europe for ataluren, a novel protein restoration remedy which the company markets as Translarna.

The US Food and Drug Administration has remained unpersuaded, however, rejecting a submission from PTC for the third time in late 2017.

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