BioMarin to Stop Developing its Duchenne Muscle Disease Drugby
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.
BioMarin plans to continue to explore other treatments called oligonucleotides for the disease, according to the statement.