BioMarin Announces Withdrawal of Market Authorization Application for Kyndrisa™ (drisapersen) in Europe
May 31 2016 - 4:05PM
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that it
has withdrawn its Kyndrisa™ (drisapersen) Marketing Authorization
Application (MAA) from the European Medicines Agency (EMA)
following discussions at the May 2016 Committee for Medicinal
Products for Human Use (CHMP) meeting. Those discussions
clearly indicated that the CHMP intended to issue a negative
opinion. Kyndrisa is an experimental drug for the treatment
of Duchenne muscular dystrophy (DMD) amenable to exon 51
skipping.
Based on discussions at the CHMP meeting and the
Food and Drug Administration Complete Response Letter in January,
BioMarin intends to discontinue clinical and regulatory development
of Kyndrisa as well as the three other first-generation follow-on
products, BMN 044, BMN 045 and BMN 053, currently in Phase 2
studies for distinct forms of Duchenne muscular dystrophy.
Notwithstanding this outcome for Kyndrisa in Europe, the Company
continues to expect to achieve non-GAAP break-even or better in
2017.
BioMarin plans to work with physicians, patient
groups, and regulatory authorities to develop a transition plan for
those patients currently being treated with Kyndrisa, BMN 044, BMN
045 and BMN 053. The Company will continue to explore the
development of next generation oligonucleotides for the treatment
of Duchenne muscular dystrophy.
“The withdrawal of the MAA and discontinuation
of our current experimental drugs for Duchenne is a difficult but
necessary decision at this time,” said Jean-Jacques Bienaimé,
BioMarin chairman and chief executive officer. Mr. Bienaimé
added, “We want to extend our sincere gratitude to all of the
families and caregivers who supported our efforts over the last
year to bring Kyndrisa to patients with Duchenne. Our plan
now is to invest in research of next generation oligonucleotides
with the goal of making a safe and effective treatment available
for boys with this devastating disorder.”
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
Duchenne 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 people with
serious and life-threatening rare disorders. 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. Information on BioMarin's website is not incorporated
by reference into this press release.
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 future development of oligonucleotides for the
treatment of DMD. 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 research
and preclinical studies related to such product candidates; the
content and timing of decisions by regulatory authorities related
to such product candidate; 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 2015 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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