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Approval delivers first pharmaceutical therapy for children and adults
with ultra-rare, life-limiting disorder
TORONTO, July 7, 2014 /CNW/ - BioMarin Pharmaceutical Inc. (NASDAQ:BMRN)
announced today that Health Canada has approved VIMIZIM(elosulfase
alfa) for long-term enzyme replacement therapy in patients with a
confirmed diagnosis of mucopolysaccharidosis IVA (MPS IVA), also known
as Morquio A syndrome. The approval makes VIMIZIM the first and only
pharmaceutical treatment option available in Canada for children and
adults living with this severely debilitating, progressive and
"Morquio A is an ultra-rare disease for which there is no cure. In fact,
until now, available treatments only addressed the symptoms of the
disease," said Dr. John Mitchell, a leading pediatric endocrinologist
and biochemical geneticist based in Montreal, and clinical investigator
in the VIMIZIM Phase 3 trial. "The approval of VIMIZIM fills a critical
unmet need for Morquio A patients and their families as it moves
treatment beyond supportive care to addressing the underlying cause of
The New Drug Submission for VIMIZIM was submitted to Health Canada in
October 2013 under Priority Review Status, which allows for the
"fast-tracking" of submissions intended for the treatment, prevention
or diagnosis of serious, life-threatening or severely debilitating
diseases or conditions.
"The Health Canada approval of VIMIZIM is a significant milestone for
BioMarin, and for Canadians living with Morquio A and their families,"
said Hank Fuchs, M.D., Chief Medical Officer of BioMarin. "To date,
BioMarin has developed treatments for three different MPS diseases. The
approval of VIMIZIM firmly establishes our leadership in advancing
important therapies for the treatment of MPS diseases. We will continue
to build on our extensive scientific and clinical knowledge of
lysosomal storage disorders to develop therapies for other rare genetic
VIMIZIM (elosulfase alfa) is an enzyme replacement therapy (ERT) for the
treatment of patients with Morquio A syndrome, or mucopolysaccharidosis
IVA (MPS IVA). VIMIZIM is the first and only ERT designed to target the
underlying cause of Morquio A - a deficiency in the enzyme
N-acetylgalactosamine-6 sulfatase (GALNS). Infused ERT with VIMIZIM
replaces deficient GALNS activity to minimize progressive
VIMIZIM is approved for the treatment of patients with Morquio A in
Canada, the United States, and the European Union.
About Morquio A
Morquio A is a disease in which people have deficient activity of an
enzyme that is essential in the breakdown and removal of the
mucopolysaccharides called keratan sulfate.1,2 The incompletely broken down mucopolysaccharides remain stored in cells
in the body causing progressive damage.2 This excessive storage causes a systemic skeletal dysplasia, short
stature, and joint abnormalities, which limit mobility and endurance.Malformation of the chest impairs respiratory function, and looseness of
joints in the neck cause spinal instability and potentially spinal cord
compression. Other symptoms may include hearing loss, corneal clouding,
and heart disease. Initial symptoms often become evident in the first
five years of life.3,4,5 The disease substantially limits both the quality and length of life of
The rate of incidence of Morquio A is as yet unconfirmed and varies
among different populations, and estimates vary between 1 in 76,000
live births and 1 in 640,000 live births.6 The incidence rate in Canada is estimated at 0.33-0.5 per 100,000 live
births.7 Globally, the estimated prevalence is approximately 3,000 patients in
the developed world.8
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: Naglazyme®
(galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly
developed and commercialized by BioMarin; Aldurazyme® (laronidase) for
mucopolysaccharidosis I (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; Firdapse®
(amifampridine), which has been approved by the European Commission for
the treatment of Lambert Eaton Myasthenic Syndrome (LEMS); and VIMIZIM
(N-acetylgalactosamine 6-sulfatase) for the treatment of Morquio A (MPS
IVA). Product candidates include: BMN 165 (PEGylated recombinant
phenylalanine ammonia lyase), also referred to as PEG-PAL, which is
currently in Phase 3 clinical development for the treatment of PKU; 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; BMN 701, a novel fusion of acid alpha glucosidase (GAA)
with a peptide derived from insulin like growth factor 2, 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 1 clinical development for the treatment of achondroplasia;
and BMN 190, a recombinant human tripeptidyl peptidase-1 (rhTPP1) for
the treatment of late-infantile neuronal ceroid lipofuscinosis (CLN2),
a form of Batten Disease.
For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference
into this press release.
VIMIZIM is our trademark, and BioMarin®, Naglazyme®, Kuvan®, Firdapse®
are registered trademarks of BioMarin Pharmaceutical Inc.
Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.
1 The Canadian Society for Mucoploysaccaride & Related Diseases Inc. A
Guide to Understanding Mucopolysaccharidosis (MPS) IV.
2 Mayo Medical Laboratories. Mucopolysaccharides (MPS) Screen, Urine.
Available at: www.mayomedicallaboratories.com/test-catalog/Clinical+and+Interpretive/84464. Accessed on February 26, 2014.
3 Borlot et al. Mucopolysaccharidosis type IVA: Evidence of primary and
secondary central nervous system involvement. American Journal of
Medical Genetics Part A. Available at: http://onlinelibrary.wiley.com/doi/10.1002/ajmg.a.36424/abstract. Access on February 26, 2014.
4 Regier, Debra S et al. Mucopolysaccharidosis Type IVA. Available at: www.ncbi.nlm.nih.gov/books/NBK148668/. Accessed on February 26, 2014.
5 Medline Plus. U.S. National Library of Medicine - National Institutes
of Health. Morquio syndrome. Available at: www.nlm.nih.gov/medlineplus/ency/article/001206.htm. Accessed on February 26, 2014.
6 Nelson J. Incidence of the mucopolysaccharidoses in Northern Ireland.
Hum Genet. 1997; 101:355-358.
7 National MPS Society. A guide to understanding MPS IV. Published 2008.
Available at: http://www.mpssociety.org/wp-content/uploads/2011/07/booklet_MPS_IV_v6.pdf. Accessed September 2012.
8 Regroupement québécois des maladies orphelines (RQMO). RQMO joins
Morquio A Community to mark International MPS Awareness Day. Available
at: http://www.newswire.ca/en/story/1356147/rqmo-joins-morquio-a-community-to-mark-international-mps-awareness-day. Accessed on: May 27, 2014.