FDA Rejects New Drug Application for BioMarin's Duchenne Treatment

BioMarin Pharmaceutical Inc. on Thursday said the U.S. Food and Drug Administration had rejected its new drug application for a treatment of a fatal form of muscular dystrophy because of questions about the drug's effectiveness.

The biopharmaceutical company said it would work with the FDA to determine next steps for the treatment, called drisapersen, and that ongoing studies will continue.

The drug, which has the marketing name Kyndrisa, remains under review in Europe.

Duchenne muscular dystrophy is a disorder occurring in about 1 in 3,500 boys and is often fatal by the time the boy reaches his 20s or early 30s. Children with Duchenne begin to lose their ability to walk in their teens, and can lose respiratory function and begin to have severe cardiac problems. It has no FDA-approved therapy.

In November, an advisory panel for the FDA said the drug's trials fell short of proving it helped children with the illness. But some panelists left open the possibility that individual children might yet respond to the drug, despite the overall negative finding.

The genetic disease is caused by a lack of a protein called dystrophin, which results in muscle fibers that degenerate and cause the patients to fall frequently. BioMarin's drug is designed to increase the production of dystrophin, which theoretically could lead to greater muscle strength and clinical benefit for the patients.

Meanwhile, the treatment is still under review for approval in Europe, and BioMarin expects the Committee for Medicinal Products for Human Use of the European Medicines Agency will make a decision in the first half of 2016. If approved, the company would receive marketing authorization for the drug in all European Union member states.

Shares of BioMarin, down 19% over the past three months, fell 2.1% to $82.35 in morning trading in New York.

Write to Anne Steele at [e-mail]

(END) Dow Jones Newswires

January 14, 2016 10:05 ET (15:05 GMT)

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