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Keeping Track: Orphan Drugs Aplenty At US FDA, From Pyrukynd Approval To Adagrasib Standard Review

 
• By Bridget Silverman

The latest drug development news and highlights from the Pink Sheet’s US FDA Performance Tracker

Keeping Track Feature image

The US Food and Drug Administration’s approval of Agios Pharmaceuticals, Inc.’s Pyrukynd (mitapivat) capped a strong week for rare diseases at the agency.

Three of the six novel agents approved in 2022 so far by the Center for Drug Evaluation and Research have...

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EMA Nod For First MASH Drug Rezdiffra, Cell Therapy Zemcelpro And 11 Others

 
• By Eliza Slawther

Madrigal Pharmaceuticals’ Rezdiffra is on track to become the first medicine approved in the EU for non-cirrhotic metabolic dysfunction-associated steatohepatitis after the European Medicines Agency recommended that it be granted conditional marketing authorization.

Woodcock: Do Not Do The Wrong Study, Even If Against US FDA Advice

 
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Highlights from Day Three of the BIO International Convention include the realities of MFN pricing, AstraZeneca's R&D plans for obesity, the need for resources for the FDA's rare disease hub and reactions to the Commissioner's National Priority Review Voucher program.

Kisunla Back In The Spotlight As EMA Reconsiders Alzheimer’s Drug Rejection

 
• By Eliza Slawther

It appears that the European Medicines Agency is making progress on its re-examination of Eli Lilly’s Alzheimer’s disease drug Kisunla, following an initial rejection in March based on safety concerns.

More from Pink Sheet

Woodcock: Do Not Do The Wrong Study, Even If Against US FDA Advice

 
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AI Will Bolster, Not Squelch, Patient Voice In Clinical Research, US FDA Says

 
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UK Looks To Auto-Match NHS Patients With Clinical Trials To Boost Recruitment

 
• By Vibha Sharma

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