BioMarin Receives Positive Opinion From the CHMP in the European Union for
VIMIZIM(TM) (elosulfase alfa) for Morquio A Syndrome
SAN RAFAEL, Calif., Feb. 20, 2014 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) announced today that the Committee for Medicinal Products
for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a
positive opinion for the company's Marketing Authorization Application (MAA)
for VIMIZIM™ (elosulfase alfa) for the treatment of Morquio A syndrome, also
called Mucopolysaccharidosis Type IVA (MPS IVA). The CHMP's recommendation is
now referred to the European Commission (EC). If approved by the EC, BioMarin
would receive marketing authorization for VIMIZIM in all EU Member States. The
EC is expected to render a final decision for VIMIZIM in the second quarter of
"This positive CHMP opinion is an important milestone in our mission to
provide the first approved therapy to treat Morquio A patients across Europe.
We will leverage our existing European infrastructure to ensure that these
patients gain access to VIMIZIM as quickly as possible," said Jean-Jacques
Bienaimé, Chief Executive Officer of BioMarin. "We are grateful for the
continuous support we have received from the Morquio A community, and in
particular, those patients who participated in the clinical development of
VIMIZIM and their families."
"As a treating physician, the CHMP opinion is meaningful news for Morquio A
patients who currently have no specific drug treatment option beyond
supportive care," said Christian J. Hendriksz, Salford Royal NHS Foundation
Trust. "VIMIZIM improved endurance in clinical trials, which may change the
course of this devastating disease."
The U.S. Food and Drug Administration (FDA) approved VIMIZIM for patients with
Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome) on February 14,
VIMIZIM is an enzyme replacement therapy for the treatment of patients with
the lysosomal storage disorder Morquio A syndrome, which is an ultra-rare,
severely debilitating disease that affects an estimated 3,000 patients in the
developed world. VIMIZIM is the first approved drug treatment for Morquio A
The disease occurs as a result of a deficiency of activity in an enzyme
involved in glycosaminoglycan (GAG) metabolism. The pervasive and progressive
accumulation of GAGs leads to significant morbidities and multisystemic
clinical impairments resulting in diminished functional capacity, impaired
quality of life, and early mortality. The most common features of the disease
are progressive skeletal dysplasia, the need for frequent surgical procedures
related primarily to musculoskeletal or respiratory dysfunction, and
significant limitations in mobility, endurance, and breathing.
The CHMP is a scientific committee composed of representatives from the
28-member states of the EU, and Iceland and Norway.The committee reviews
medical product applications on their scientific and clinical merit and
provides advice to the EC, which has the authority to approve medicines for
the EU.The EU, which generally follows the recommendation of the CHMP, is
expected to make its final decision in about 60 days.
VIMIZIM (elosulfase alfa) is a treatment for patients with Morquio A syndrome,
or mucopolysaccharidosis IVA (MPS IVA).VIMIZIM is the first enzyme
replacement therapy (ERT) designed to target the underlying cause of Morquio A
Syndrome – a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase
(GALNS). VIMIZIM is intended to provide the exogenous enzyme GALNS that will
be taken up into the lysosomes and increase the catabolism of GAGs.Morquio A
syndrome is a rare, severely debilitating and progressive disease that
previously had no standard accepted treatment other than supportive care.
About Morquio A Syndrome
Morquio A syndrome, or Mucopolysaccharidosis IVA (MPS IVA) is a disease in
which people are missing an enzyme that is essential in the breakdown and
removal of the glycosaminoglycans (GAGs) called keratan sulfate (KS) and
chondroitin-6-sulfate (C6S). The incompletely broken down GAGs remain stored
in cells in the body causing progressive damage. This excessive storage causes
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.The disease substantially limits both the
quality and length of life of those affected.
The rate of incidence of Morquio A syndrome is as yet unconfirmed and varies
among different populations, and estimates vary between 1 in 200,000 live
births and 1 in 450,000 live births.
Important Safety Information
Life-threatening allergic reactions, known as anaphylaxis, can occur during
VIMIZIM^TM (elosulfase alfa) infusions. Due to the potential for anaphylaxis,
appropriate medical support should be readily available when VIMIZIM is
administered and for an appropriate period of time following administration.
Hypersensitivity reactions have been observed as early as 30 minutes after the
start of infusion but as late as six days after infusion. Frequent symptoms of
hypersensitivity reactions included anaphylactic reactions, urticaria,
peripheral edema, cough, dyspnea, and flushing.
Because of the potential for hypersensitivity reactions, administer
antihistamines with or without antipyretics prior to infusion.If severe
hypersensitivity reactions occur, immediately stop the infusion of VIMIZIM and
initiate appropriate treatment. Patients with acute febrile or respiratory
illness at the time of VIMIZIM infusion may be at higher risk of
life-threatening complications from hypersensitivity reactions.
Sleep apnea is common in MPS IVA patients. Evaluation of airway patency should
be considered prior to initiation of treatment with VIMIZIM. Patients using
supplemental oxygen or continuous positive airway pressure (CPAP) during sleep
should have these treatments readily available during infusion in the event of
an acute reaction, or extreme drowsiness/sleep induced by antihistamine use.
Spinal or cervical cord compression (SCC) is a known and serious complication
of MPS IVA and may occur as part of the natural history of the disease. In
clinical trials, SCC was observed both in patients receiving VIMIZIM and
patients receiving placebo. Patients with MPS IVA should be monitored for
signs and symptoms of SCC (including back pain, paralysis of limbs below the
level of compression, urinary and fecal incontinence) and given appropriate
All patients treated with VIMIZIM 2 mg/kg once per week in the
placebo-controlled trial developed anti-drug antibodies.
VIMIZIM should be used during pregnancy only if the potential benefit
justifies the potential risk to the fetus. It is not known if VIMIZIM is
present in human milk.
Safety and effectiveness in pediatric patients below 5 years of age have not
In clinical trials, the most common adverse reactions (≥10%) occurring during
infusion included pyrexia, vomiting, headache, nausea, abdominal pain, chills,
and fatigue. The acute reactions requiring intervention were managed by either
temporarily interrupting or discontinuing infusion, and administering
additional antihistamine, antipyretics, or corticosteroids.
Please see full Prescribing Information, including boxed warning, or visit
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;
Naglazyme® (galsulfase) for MPS VI; Aldurazyme® (laronidase) for MPS I, a
product which BioMarin developed through a 50/50 joint venture with Genzyme, a
Sanofi Company; Kuvan® (sapropterin dihydrochloride) 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 PEG PAL (PEGylated
recombinant phenylalanine ammonia lyase), 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 protein of insulin-like growth factor 2 and acid alpha glucosidase
(IGF2-GAA), which is currently in Phase 1/2clinical 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, BMN 190, a recombinant human tripeptidyl peptidase-1 (rhTPP1)
for the treatment of late-infantile neuronal ceroid lipofuscinosis (CLN2), 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.
This press release contains forward-looking statements about the business
prospects of BioMarin Pharmaceutical Inc., including, without limitation,
statements about: expectations regarding the approval of VIMIZIM by the FDA
and the EMA, BioMarin's ability to support the launch of a new product and
ship to specialty pharmacies, the company's ability to conduct additional
clinical trials. 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: the
results and timing of actions by the FDA and the EMA, results and timing of
current and planned clinical trials of its products; the timing of pricing
approval in several countries in Europe; the availability and launch of
VIMIZIM in Europe and the market potential for VIMIZIM as a treatment for
Morquio A, 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 2012 Annual Report on
Form 10-K, and the factors contained in BioMarin's reports on Form
10-Q.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.
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.
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