BioMarin Posts Presentation From FDA Advisory Committee Meeting for Kyndrisa(TM) (drisapersen) for the Treatment of Duchenne ...
November 24 2015 - 10:04AM
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the
Company’s presentation from the Peripheral and Central Nervous
System Drugs Advisory Committee of the U.S. Food and Drug
Administration (FDA)’s meeting for KyndrisaTM (drisapersen) is now
available. The Advisory Committee is meeting today to discuss the
data submitted to support the New Drug Application (NDA) for
Kyndrisa for the treatment of Duchenne muscular dystrophy amenable
to exon 51 skipping.
Interested parties may access BioMarin’s presentation from
today’s Advisory Committee meeting via the link below to the
investor section of the BioMarin website.
http://investors.bmrn.com/common/download/download.cfm?companyid=ABEA-3W276N&fileid=863346&filekey=AEF00248-5747-4ACF-8C2F-39F5B1D7FCC3&filename=BMRN_AdCom_with_safe_harbor_112415.pdf
About Duchenne Muscular Dystrophy
Changes 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. Dystrophin protein plays an
important structural role in the performance of muscles. Without
dystrophin, 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 Skipping
Kyndrisa 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.
Kyndrisa is the first and only investigational medicine designed
specifically for the treatment of Duchenne that has received orphan
drug, breakthrough drug, fast track and priority review status by
the FDA. The Kyndrisa clinical development program is the
largest ever submitted to the FDA for the condition and includes
more than 300 Duchenne patients and multiple randomized
placebo-controlled studies.
About BioMarin
BioMarin 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.
Kyndrisa™ is our trademark, and BioMarin® is a registered
trademark of BioMarin Pharmaceutical Inc.
Investors:
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558
Media:
Debra Charlesworth
BioMarin Pharmaceutical Inc.
(415) 455-7451
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