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Cover Story: Regulation: Not so fast

Why approval of Exondys 51 might be a setback for patient-focused regulation

By Steve Usdin, Washington Editor

FDA documents concerning the review of Exondys 51 eteplirsen to treat DMD show that the chorus keening that the approval opens the floodgates for companies to combine shoddy, underpowered studies with patient activism to gain easy approvals and quick riches has misinterpreted the episode.

The Duchenne muscular dystrophy community's interaction with FDA had been hailed as a model for patient-focused drug development, and the accelerated approval of Sarepta Therapeutics Inc.'s NDA was a resounding victory for DMD advocates.

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    Not so fast

    Approval of Exondys 51 was both a resounding victory for DMD advocates, and a likely setback for patient-focused regulation.

  • How much is enough?

    FDA's Woodcock and Unger disagreed on one fundamental question about Exondys 51: how much dystrophin expression predicts clinical benefit in DMD?

  • Digging into dosing

    Sarepta's scanty clinical program gives small clues to choosing a dose of Exondys 51 that will produce enough dystrophin.

  • Wrong again

    If the $300,000 price tag Sarepta put on Exondys limits the drug's use to a fraction of DMD patients, there was no point to accelerated approval.


    On NPV, DCF and IRR(elevance)?

    Steven Holtzman on why judgment trumps financial analysis in evaluating a deal.

  • Ebb & Flow

    Tobira's 500% payout. Plus: iOmx checks in on cancer cells; and Artios' plans for a DDR empire.

  • THE DISTILLERY brings you this week's most essential scientific findings in therapeutics, distilled by Innovations editors from a weekly review of more than 400 papers in 41 of the highest-impact journals in the fields of biotechnology, the life sciences and chemistry. The Distillery goes beyond the abstracts to explain the commercial relevance of featured research, including licensing status and companies working in the field, where applicable.

    This week in therapeutics includes important research findings on targets and compounds, grouped first by disease class and then alphabetically by indication.

    This week in techniques includes findings about research tools, disease models and manufacturing processes that have the potential to enable or improve all stages of drug discovery and development.

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