BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced
completion of the rolling submission of a New Drug Application
(NDA) to the United States Food and Drug Administration (FDA) for
drisapersen, an investigational exon-skipping drug candidate for
the treatment of the largest genetically defined subset of Duchenne
muscular dystrophy (DMD). DMD is the most common fatal genetic
disorder diagnosed in childhood, affecting approximately 1 in every
3,500 live male births with about 20,000 new cases diagnosed
globally each year. Drisapersen induces the skipping of dystrophin
exon 51, potentially providing a therapeutic benefit to DMD
patients for whom skipping of exon 51 restores the proper
dystrophin reading frame, corresponding to approximately 13% of DMD
patients. The company intends to also submit an application for
registration in the European Union in summer 2015.
"We believe drisapersen may offer a meaningful benefit to boys
living with DMD whose mutations are amenable to exon 51 skipping.
The totality of data on drisapersen contains three randomized,
placebo-controlled, efficacy trials and two long term extension
studies, which include some boys treated for approximately 3.4
years," said Camilla V. Simpson, Global Head of Regulatory Affairs,
Pharmacovigilance. "With this application, BioMarin continues in
its long-standing tradition of developing important therapies for
those who are most in need. The submission of the NDA
represents a significant milestone for BioMarin, and we appreciate
the strong, collaborative effort of many hard working employees,
investigators, patients and their families. We look forward to
working with the U.S. Regulatory Authorities to thoroughly
understand the data generated for this heterogenous and critically
ill patient population and hopefully to bring this treatment to
patients expeditiously."
Drisapersen has been granted Orphan and Fast Track status, as
well as Breakthrough Therapy designation by the FDA.
DMD is caused by a mutation in the gene that encodes
for dystrophin, a protein that is important in connecting the
cytoskeleton of muscle fibers to the extracellular matrix. Its
deficiency in DMD leads to progressive muscle weakness, loss of
ambulation in early adolescence, and typically death due to
pulmonary or cardiac insufficiency in the late twenties. Because
the Duchenne gene is found on the X-chromosome, it primarily
affects boys; however, it occurs across all races and
cultures. There is currently no approved therapy in the United
States for DMD.
"This is a first for the Duchenne community, and we are filled
with hope that there could be a treatment for Duchenne in the
United States," said Debra Miller, co-founder and CEO of
CureDuchenne. "CureDuchenne has been supporting the development of
drisapersen for more than a decade, and we are delighted that
BioMarin has reached this important stage. We salute the
researchers who have been working so hard, and we share their
determination to find a cure for Duchenne."
About Drisapersen
Duchenne muscular dystrophy (DMD) is a severely
debilitating childhood neuromuscular disease that affects up to 1
in 3,500 live male births. This rare disease is caused by mutations
in the dystrophin gene, resulting in the absence or defect of the
dystrophin protein. As a result, patients suffer from progressive
loss of muscle strength, often rendering them wheelchair-bound
before the age of 12 years. Respiratory and cardiac muscle can also
be affected by the disease and most patients die in early adulthood
due to respiratory and cardiac failure.
About Exon Skipping
Exons are the parts of a gene that contain the
instructions for generating a protein. In DMD, mutations in the
dystrophin gene lead to the absence of dystrophin protein,
resulting in the most severe form of muscular dystrophy. In
applicable cases, skipping an exon near the mutation allows for the
production of a truncated but functional dystrophin protein.
About BioMarin
BioMarin develops and commercializes innovative
biopharmaceuticals for serious diseases and medical conditions. The
company's product portfolio comprises five approved products and
multiple clinical and pre-clinical product candidates. Approved
products include Vimizim® (elosulfase alfa) for MPS IVA, a product
wholly developed and commercialized by BioMarin; Naglazyme®
(galsulfase) for MPS VI, a product wholly developed and
commercialized by BioMarin; Aldurazyme® (laronidase) for MPS I, a
product which BioMarin developed through a 50/50 joint venture with
Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Powder
for Oral Solution and Tablets, for phenylketonuria (PKU), developed
in partnership with Merck Serono, a division of Merck KGaA of
Darmstadt, Germany and Firdapse® (amifampridine), which has been
approved by the European Commission for the treatment of Lambert
Eaton Myasthenic Syndrome (LEMS). Product candidates include
drisapersen, an exon skipping oligonucleotide, for which a
marketing application has been submitted to FDA for the treatment
of patients with Duchenne muscular dystrophy (DMD) with mutations
in the dystrophin gene that are amenable to treatment with exon 51
skipping, pegvaliase (PEGylated recombinant phenylalanine ammonia
lyase, formerly referred to as BMN 165 or PEG PAL), which is
currently in Phase 3 clinical development for the treatment of PKU,
talazoparib (formerly referred to as BMN 673), a poly ADP-ribose
polymerase (PARP) inhibitor, which is currently in Phase 3 clinical
development for the treatment of germline BRCA breast cancer,
reveglucosidase alfa (formerly referred to as BMN 701), a novel
fusion protein of insulin-like growth factor 2 and acid alpha
glucosidase (IGF2-GAA), which is currently in Phase 3 clinical
development for the treatment of Pompe disease, BMN 111, a modified
C-natriuretic peptide, which is currently in Phase 2 clinical
development for the treatment of achondroplasia, BMN 044, BMN 045
and BMN 053, exon skipping oligonucleotides, which are currently in
Phase 2 clinical development for the treatment of Duchenne muscular
dystrophy (exons 44, 45 and 53), cerliponase alfa (formerly
referred to as BMN 190), a recombinant human tripeptidyl
peptidase-1 (rhTPP1) for the treatment of CLN2 disorder, a form of
Batten disease, which is currently in Phase 1, BMN 270, an
AAV-factor VIII vector, for the treatment of hemophilia A and BMN
250, a novel fusion of alpha-N-acetyglucosaminidase (NAGLU) with a
peptide derived from insulin-like growth factor 2 (IGF2), for the
treatment of MPS IIIB.
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
rolling NDA submission for drisapersen with the FDA and the pending
submission to the European Medicines Agency (EMA); the potential
outcome of the review of such filings; and the possible approval of
such product candidates. 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 its product candidates;
the content and timing of decisions by the FDA, the EMA and other
regulatory authorities concerning its product candidates; 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.
BioMarin®, Naglazyme®, Kuvan®, Firdapse® and VIMIZIM® are
registered trademarks of BioMarin Pharmaceutical Inc.
Aldurazyme® is a registered trademark of BioMarin/Genzyme
LLC.
CONTACT: Investors:
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558
Media:
Debra Charlesworth
BioMarin Pharmaceutical Inc.
(415) 455-7451
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