FDA Rejects New Drug Application for BioMarin's Duchenne Treatment
January 14 2016 - 10:20AM
Dow Jones News
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 Anne.Steele@wsj.com
(END) Dow Jones Newswires
January 14, 2016 10:05 ET (15:05 GMT)
Copyright (c) 2016 Dow Jones & Company, Inc.
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