BioMarin Kills Kyndrisa, But Duchenne Pursuit Not Over
This article was originally published in The Pink Sheet Daily
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.
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PTC Therapeutics appears to be hoping that CDER Director Woodcock will do for ataluren what she did for Sarepta's exon-skipping Duchenne muscular dystrophy treatment Exondys 51 since dispute resolution request would first go to Office of New Drugs, where Woodcock is acting director.
PTC Therapeutics argues FDA did not apply the same standard to Translarna in Duchenne muscular dystrophy as previous DMD products, but FDA says accelerated approval was not possible because of negative efficacy data.
Several members of the Peripheral and Central Nervous System Drugs Advisory Committee meeting looking at PTC' Therapeutics' proposed Duchenne muscular dystrophy treatment also looked at Sarepta's Exondys 51 and BioMarin's Kyndrisa, but most did not vote or comment favorably.