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Sarepta Smacked By FDA's Curiously Timed Compassionate Use Revisions

Executive Summary

Sarepta Therapeutics Inc. investors panicked over the FDA's June 2 release of revised expanded access guidances – worried the action was in preparation for the agency to reject the company's Duchenne muscular dystrophy drug eteplirsen, whose verdict was curiously delayed last week.

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BioMarin Kills Kyndrisa, But Duchenne Pursuit Not Over

Seeing no clear path to approval in the US or Europe for its Duchenne muscular dystrophy drug Kyndrisa (drisapersen), BioMarin Pharmaceutical Inc. has decided to shelve the experimental medicine, along with three other first-generation follow-on products. But it said its Duchenne pursuit is not completely over, declaring it continues to "explore" its early-stage next-generation oligonucleotides as potential treatments for the disease, which generally kills patients before they reach 30 years.

Limbo Land For Sarepta, Duchenne And Rare Diseases

While investors were hopeful the FDA's delay in issuing its verdict for Sarepta Therapeutics Inc.'s Duchenne muscular dystrophy medicine eteplirsen may mean the agency is not prepared to reject it, the postponement may create more doubt and uncertainty about the path forward in rare disease drug development in general.

FDA Refuses To File Translarna NDA; PTC Plummets

Just as things looked like they couldn't get any worse for the Duchenne muscular dystrophy (DMD) research and development community – and for patients – PTC Therapeutics Inc. revealed it had received a refuse-to-file (RTF) letter from the FDA for the firm's new drug application (NDA) for Translarna (ataluren).

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