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BioMarin Kills Kyndrisa, But Duchenne Pursuit Not Over

 
• By Donna Young

Seeing no clear path to approval in the US or Europe for its muscular dystrophy drug drisapersen, BioMarin shelves it , plus three first-generation follow-on products. But it continues to "explore" its early stage next-generation oligonucleotides as potential treatments for DMD.

With the FDA already rejecting BioMarin Pharmaceutical Inc.'s experimental Duchenne muscular dystrophy (DMD) drug Kyndrisa (drisapersen) and now European regulators indicating they saw no chance for success, the biotech said it was ending development of the drug, plus three other first-generation follow-on products – BMN 044, BMN 045 and BMN 053 – which currently are in Phase II studies for distinct forms of the disease.

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Ocaliva: Still No Clarity On Why EU Court Opposed Revocation Of Approval

 
• By Neena Brizmohun

Advanz Pharma would have had to show that the European Commission’s decision to revoke Ocaliva’s conditional marketing approval risked causing serious and irreparable harm, according to lawyers from Van Bael & Bellis.

Final Chance To Have Your Say: Take Our Reader Survey This Week

 
• By Nielsen Hobbs

This is your final call to participate in the survey to better understand our subscribers’ content and delivery needs. The deadline is 20 September.

Shape Our Content: Take The Reader Survey

 
• By Nielsen Hobbs

We are conducting a survey to better understand our subscribers’ content and delivery needs. If there are any changes you’d like to see in coverage topics, article format, or the method in which you access the Pink Sheet – or if you love it how it is – now is the time to have your voice heard.

Brazil Pilots Digital Drug Pack Inserts

 
• By Francesca Bruce

A new pilot aims to take Brazil closer to ‘digital transformation.’

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EU Pharmacopoeia Adopts ‘Groundbreaking’ Cell Therapy Chapter, Seeks Feedback On Alternative Microbiological Tests

 
• By Neena Brizmohun

A new chapter that will be published in the European Pharmacopoeia soon is expected to provide a comprehensive framework for the production and quality control of cell-based preparations. Meanwhile, an existing chapter has been revised to facilitate the implementation of rapid microbiological testing methods.

US FDA User Fee Collection Safe In Preliminary Trump 2026 Budget Plan

 
• By Cathy Kelly

The preliminary White House 2026 budget plan cuts agency funding, but not so sharply that user fees are excluded from collection.

Beyond Tariffs: The Silver Lining Of The US Pharma Security Investigation

 
• By Jessica Merrill

An investigation by the US Secretary of Commerce into pharmaceutical imports gives industry an opportunity to comment. AAM CEO John Murphy talked to Pink Sheet's sister publication Scrip about the latest developments.