FDA Issues Complete Response Letter for KyndrisaTM for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
January 14 2016 - 8:30AM
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the
U.S. Food and Drug Administration (FDA) issued a Complete Response
letter to the Company’s New Drug Application (NDA) for KyndrisaTM
(drisapersen) for the treatment of Duchenne muscular dystrophy
(Duchenne) amenable to exon 51 skipping.
The FDA issues Complete Response letters to indicate that the
review cycle for an application is complete and that the
application is not ready for approval in its present form.
FDA has concluded that the standard of substantial evidence of
effectiveness has not been met. BioMarin is reviewing the Complete
Response Letter and will work with the FDA to determine the
appropriate next steps regarding this application.
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.
The ongoing Kyndrisa extension studies will continue, as will
the ongoing clinical trials for other exon-skipping
oligonucleotides, BMN 044, BMN 045 and BMN 053, while BioMarin is
exploring next steps for this application. Patients currently
receiving Kyndrisa, BMN 044, BMN 045 and BMN 053 will remain on
therapy.
Kyndrisa Marketing Authorization Application Remains
Under Regulatory Review in Europe
An application for marketing approval of Kyndrisa is also under
review in the European Union. BioMarin anticipates that the
Committee for Medicinal Products for Human Use (CHMP) of the
European Medicines Agency (EMA) will provide an opinion for the
company's Marketing Authorization Application (MAA) for Kyndrisa
for the treatment of Duchenne muscular dystrophy (Duchenne)
amenable to exon 51 skipping in the first half of 2016. If the CHMP
opinion is positive, the MAA will be referred to the European
Commission (EC). If the MAA is approved by the EC, BioMarin
would receive marketing authorization for Kyndrisa in all EU Member
States. The EC is expected to render a final decision for Kyndrisa
in the second half of 2016.
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.
About Duchenne Muscular
Dystrophy
Duchene muscular dystrophy is an x-linked
genetic disorder that affects mostly boys. In Duchenne,
boys begin to show signs of muscle weakness as early as two to
five years of age. The disease gradually weakens the skeletal or
voluntary muscles in the arms, legs and trunk. Due to progressive
muscle weakness, Duchenne patients are often wheelchair
bound between the ages of seven and 13 years old. At a later
stage, the boys’ respiratory and cardiac muscles are also affected
and for most boys, respiratory and cardiac failure are major causes
of death, often prevalent by the age of 20.
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.
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 possible path forward to approve Kyndrisa in the
United States and the review of the marketing authorization
application in Europe. 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, the EMA 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.
Contacts:
Investors
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558
Media
Debra Charlesworth
BioMarin Pharmaceutical Inc.
(415) 455-7451
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