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Keeping Track: Another Gene Therapy Approval, Several Priority Review Designations, And Many Resubmissions

 
• By Michael Cipriano and Sue Sutter

The latest drug development news and highlights from our US FDA Performance Tracker.

Keeping Track Feature image

Here's your news in brief: Gene therapies are continuing to make their way onto the scene, with Kite Pharma Inc.'s chimeric antigen receptor T-cell (CAR-T) therapy Yescarta (axicabtagene ciloleucel) becoming the second gene therapy to win a US FDA approval.

Pacira Pharmaceuticals Inc.and Charleston Laboratories Inc. are both working their way back from complete response letters (CRLs) for...

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• By Sue Sutter

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• By Bridget Silverman

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US FDA To Advisory Committee Industry Reps: Don’t Talk Too Much

 
• By Sue Sutter

Nonvoting industry representative’s presence at Pediatric Advisory Committee meeting was required by statute, the agency said, a de facto acknowledgement that an April directive precluding industry reps from sitting on advisory committees has had little practical effect.

Capricor Gets Complete Response For DMD Cell Therapy: Unconvincing Data Or New Attitude At FDA?

 
• By Joseph Haas

Seven weeks before its action date and a month after an advisory committee was hastily cancelled, Capricor gets a CRL for its cell therapy deramiocel in DMD-related cardiomyopathy citing efficacy and manufacturing concerns. It has a Phase III study nearing readout, though.

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Animal Testing Alternatives: US Pharmacopeia On Its Testing Transition

 
• By Eliza Slawther

In this second of a two-part series of articles, US Pharmacopeia CEO Ronald T. Piervincenzi tells the Pink Sheet which animal testing models have alternative options and highlights where further investment and research is needed to develop appropriate alternatives.

EU Industry Proposes 3-Basket Strategy To Phase Out Animal Testing

 
• By Eliza Slawther

A “basket approach” to phasing out animal testing could help identify areas where “moonshot” research is required to implement new approach methodologies, according to EU industry federation EFPIA.

England Leads Europe In Reimbursing Vertex’s Next-In-Class CF Drug Alyftrek

 
• By Neena Brizmohun

Vertex said it reached “a broad reimbursement agreement” with the National Health Service for its once-daily, triple combination treatment for cystic fibrosis. As for reimbursement in the rest of Europe, the company is targeting Ireland, Denmark and Germany next.