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When Breakthrough Falls Short: US FDA Complete Response Letters Rising Among BTD Applications For Approval

 
• By Bridget Silverman

More than half of the complete response letters issued for applications holding breakthrough status have been issued since 2020, reflecting difficult – and often rancorous – efficacy debates.

Breakthrough designations have allowed many therapies to climb quickly through development and review, but more are falling short of approval and getting complete response letters. • Source: Alamy

Complete response letters are becoming a more likely outcome for applications with the FDA’s marquee expedited review status, an analysis using the Pink Sheet’s US FDA Performance Tracker shows.

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Political Official Pushing Novavax To Conduct COVID-19 Vaccine Postmarket RCT

 
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In an unusual move, Tracy Beth Høeg, a special assistant to FDA Commissioner Martin Makary, is leading the continued negotiations on Novavax’s delayed COVID-19 vaccine approval.

Mesoblast’s Ryoncil: US FDA Changed Its Mind On Need For A Randomized Trial

 
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Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.

Ryoncil Chronology: Three Review Cycles, Two CRLs, One Dispute Resolution Request

 
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The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.

Pink Sheet Podcast: More US FDA Departures, Makary On Combined AE Database, ‘Conditional’ Approval

 
• By Derrick Gingery, Sarah Karlin-Smith, and Sue Sutter

Pink Sheet reporter and editors discuss the most recent senior staff departures at the FDA and their impact on the agency, as well as Commissioner Martin Makary’s plans for a new approval pathway and a combined adverse event database as outlined in an interview with a podcast host.

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Pink Sheet Podcast: More US FDA Departures, Makary On Combined AE Database, ‘Conditional’ Approval

 
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Vertex’s Alyftrek For CF Among Six Orphans On Track For Pan-EU Approval

 
• By Eliza Slawther

The European Medicines Agency has recommended 16 drugs for EU-wide approval, including Vertex’s cystic fibrosis drug Alyftrek and five other orphans.

Quotable: Top Experts On Policy Hot Topics

 
• By Anabel Costa-Ferreira

The Pink Sheet highlights recent comments and insights from pharma officials and executives on key issues we are covering.