BioMarin to Stop Developing its Duchenne Muscle Disease Drug

  • European regulators poised to give Kyndrisa a negative opinion
  • Company will continue to research new Duchenne treatments

BioMarin Pharmaceutical Inc. plans to stop developing its lead treatment candidate for a deadly muscle disease as European regulators signaled they’ll give it a negative review, four months after the drug was rejected in the U.S.

With the looming European denial, the decision to discontinue the development of the treatment, called Kyndrisa, and three related medications, was “difficult but necessary,” Chief Executive Officer Jean-Jacques Bienaimé said in a statement Tuesday.

There are no approved treatments for Duchenne muscular dystrophy, or DMD, a deadly genetic disease that usually affects young boys and has proven a tough disease for drugmakers working to treat it. The U.S. Food and Drug Administration delayed last week its decision on another experimental drug, made by Sarepta Therapeutics Inc.

Kyndrisa was rejected by the FDA in January, after BioMarin failed to show the drug was effective. In February, the agency rejected a DMD therapy from PTC Therapeutics Inc.

BioMarin plans to continue to explore other treatments called oligonucleotides for the disease, according to the statement.

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