FDA Posts Briefing Documents for Advisory Committee Meeting to Review Kyndrisa™ (drisapersen) for the Potential Treatment o...
November 20 2015 - 8:10AM
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that the
U.S. Food and Drug Administration (FDA) posted briefing materials
prepared by BioMarin and by the FDA. The materials pertain to the
November 24, 2015 Peripheral and Central Nervous System Drugs
Advisory Committee (PCNS) meeting to review the New Drug
Application (NDA) for Kyndrisa™ (drisapersen) for the potential
treatment of Duchenne muscular dystrophy (Duchenne) amendable to
exon 51 skipping.
The PCNS advisory meeting is scheduled for
November 24 at 8:00 a.m. to 5:30 p.m. EST. The briefing materials
and webcast information can be found on the FDA website at:
http://www.fda.gov/AdvisoryCommittees/Calendar/ucm467180.htm
The Prescription Drug User Fee Act (PDUFA)
action date for completion of FDA review of the Kyndrisa NDA is
December 27, 2015.
About Duchenne Muscular
DystrophyChanges in the dystrophin gene (mutations) that
lead to the near absence of dystrophin protein result in the most
severe form of dystrophin deficient muscular dystrophy, Duchenne
muscular dystrophy, also known as just Duchenne. Boys living
with Duchenne experience progressive muscle weakness, causing
serious medical complications including serious heart or
respiratory-related complications, resulting in death in early
adulthood.
Primarily affecting boys, Duchenne affects
approximately 1 in every 3,500-5,000 male children, making it the
most common fatal genetic disorder diagnosed in childhood.
There is currently no FDA approved therapy
designed specifically to treat Duchenne.
About Kyndrisa
and Exon SkippingKyndrisa is an antisense
oligonucleotide that induces exon skipping to provide a molecular
patch for dystrophin transcripts produced by certain mutated
dystrophin genes. Exons are the parts of a gene that contain
the instructions for generating a protein. In applicable cases,
skipping an exon near the mutation allows for the production of a
truncated but functional dystrophin protein.
About BioMarinBioMarin is a
global biotechnology company that develops and commercializes
innovative therapies for patients with serious and life-threatening
rare and ultra-rare genetic diseases. The company's portfolio
consists of five commercialized products and multiple clinical and
pre-clinical product candidates. For additional information, please
visit www.BMRN.com.
Forward-Looking Statement This
press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc., including,
without limitation, statements about: expectations regarding the
FDA’s review of the Kyndrisa NDA outcomes of the review of such
filings; and the possible approval of Kyndrisa. These
forward-looking statements are predictions and involve risks and
uncertainties such that actual results may differ materially from
these statements. These risks and uncertainties include, among
others: results and timing of current and planned clinical trials
of Kyndrisa; the content and timing of decisions by the FDA, and
other regulatory authorities concerning Kyndrisa; and those factors
detailed in BioMarin's filings with the Securities and Exchange
Commission, including, without limitation, the factors contained
under the caption "Risk Factors" in BioMarin's 2014 Annual Report
on Form 10-K, as amended, and the factors contained in BioMarin's
reports on Form 8-K. Stockholders are urged not to place undue
reliance on forward-looking statements, which speak only as of the
date hereof. BioMarin is under no obligation, and expressly
disclaims any obligation to update or alter any forward-looking
statement, whether as a result of new information, future events or
otherwise.
Kyndrisa™ is our trademark, and BioMarin® is a
registered trademark of BioMarin Pharmaceutical Inc.
Contact:
Investors:
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558
Media:
Debra Charlesworth
BioMarin Pharmaceutical Inc.
(415) 455-7451
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