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FDA’s DMD Decisions: Glass Two-Thirds Full?

 
• By Michael McCaughan

The outcome of an US advisory committee review of PTC’s ataluren was not surprising. That is bad news for PTC’s prospects of launching the drug in the near term – but given the recent history of the DMD class, it is probably good news for biopharma companies overall as a sign that FDA's neurology group is moving forward effectively.

glass half empty or half full
Half what? • Source: Kalyan Chakravarthy

In any other circumstance, the US FDA's Peripheral & CNS Drugs Advisory Committee’s Sept. 28 meeting on PTC Therapeutics Inc.’s ataluren would have been one that most sponsors could afford to skip since the briefing documents made clear the agency didn't believe ataluren’s efficacy had been demonstrated in the two trials completed by PTC to date.

PTC isn’t the first sponsor to try the equivalent of a Hail Mary pass to save an application by forcing...

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More from US FDA Performance Tracker

US FDA’s Second Half 2025 User Fee Goals Swing Toward Respiratory Disease

 
• By Bridget Silverman

The 43 novel agents with user fee goal dates in the remainder of 2025 have fewer oncologics and infectious disease drugs than the first half of the year.

Slowdown: US FDA’s 2025 Novel Approvals Are On Below-Average Pace

 
• By Bridget Silverman

US FDA's 25 novel approvals in 2025 fell short of the agency's five-year average count, driven by a drop in drugs center approvals; 45 user fee goals remain in second half of year.

US FDA’s CRL Release More Incremental Than ‘Radical’ Transparency

 
• By Bridget Silverman

The transparency initiative collects CRLs already made public in approval packages on its drugs@fda site to post on the openFDA public portal.

America Next: Most Novel Agents With July Goal Dates Have Been Approved Overseas

 
• By Bridget Silverman

The US FDA will not be in its usual position as the first regulator to approve novel drugs, with only one of the six novel candidates on the July user fee calendar seeking its first approval worldwide in the US

More from Regulatory Trackers

America Next: Most Novel Agents With July Goal Dates Have Been Approved Overseas

 
• By Bridget Silverman

The US FDA will not be in its usual position as the first regulator to approve novel drugs, with only one of the six novel candidates on the July user fee calendar seeking its first approval worldwide in the US

EU CHMP Opinions And MAA Updates

 
• By Neena Brizmohun

This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.

Global Pharma Guidance Tracker - May 2025

 
• By Vibha Sharma and Anabel Costa-Ferreira

Stay up to date on regulatory guidelines from around the world with the Pink Sheet’s Guidance Tracker. The complete Global Pharma Guidance Tracker, with sortable and searchable listings going back to 2014, is available online.