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BioMarin Kills Kyndrisa, But Duchenne Pursuit Not Over

 
• By Donna Young
  
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Seeing no clear path to approval in the US or Europe for its Duchenne muscular dystrophy drug Kyndrisa (drisapersen), BioMarin Pharmaceutical Inc. has decided to shelve the experimental medicine, along with three other first-generation follow-on products. But it said its Duchenne pursuit is not completely over, declaring it continues to "explore" its early-stage next-generation oligonucleotides as potential treatments for the disease, which generally kills patients before they reach 30 years.

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.

BioMarin CEO Jean-Jacques Bienaimé said the decision to terminate the R&D programs for the investigational DMD drugs was "difficult but...

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