BioMarin Announces FDA Approval for VIMIZIM(TM) (elosulfase alfa) for the
Treatment of Patients With Morquio A Syndrome
VIMIZIM is the First and Only Specific Treatment for Patients With This
Ultra-Rare Genetic Condition
SAN RAFAEL, Calif., Feb. 14, 2014 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) today announced that the U.S. Food and Drug Administration
(FDA) has approved VIMIZIM™ (elosulfase alfa) for patients with
Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
"The FDA approval of VIMIZIM is an important milestone for BioMarin and for
patients with Morquio A syndrome. VIMIZIM is the first and only therapy
designed to address the condition at the cellular level, fulfilling a large
unmet medical need for patients and their families," said Jean-Jacques
Bienaimé, Chief Executive Officer of BioMarin. "With the approval of VIMIZIM,
BioMarin firmly establishes its leadership in advancing therapies to treat MPS
diseases. We have developed three therapies to treat three different MPS
diseases and continue to build on our extensive scientific and clinical
knowledge of lysosomal storage disorders to develop therapies for other rare
VIMIZIM is an enzyme replacement treatment for Morquio A syndrome, which
affects an estimated 3,000 patients in the developed world. 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.
"In clinical trials, VIMIZIM was shown to significantly improve endurance,
which possibly could change the course of the disease. As a treating
physician, I am encouraged that the therapy has proven to provide clinical
benefit, which is not always possible to demonstrate with ultra-rare
diseases," said Paul Harmatz, M.D., Associate in Gastroenterology and
Nutrition at the Children's Hospital and Research Center in Oakland,
California and clinical investigator in the VIMIZIM Phase 3 trial. "The
approval of VIMIZIM is an important advance for Morquio A patients and their
families and moves treatment beyond supportive care to treating the underlying
cause of the disease."
"We are thrilled that patients with Morquio A syndrome will have access to
this potentially life-changing therapy and appreciate BioMarin's commitment to
the MPS community and the individuals and their families who are affected by
these devastating conditions," said Barbara Wedehase, MSW, CGC, Executive
Director of the National MPS Society. "Until now, patients with Morquio A
syndrome didn't have a drug treatment option. This approval provides the
community with a therapy and with hope."
Shipments of VIMIZIM to the distribution channels will commence immediately,
and BioMarin will begin promotion of VIMIZIM in the U.S. immediately.BioMarin
has also submitted marketing applications for VIMIZIM in the European Union,
Brazil, Australia, Canada, and Mexico.
BioMarin will offer support to patients through its BioMarin Patient &
Physician Support (BPPS) team.Through BPPS, patients receive live,
personalized support by a specialized case manager who will research insurance
coverage and alternative benefit options. BPPS will help patients obtain
coverage and minimize out-of-pocket expenses and find alternative financial
assistance for treatment. To reach a BPPS case manager, please call,
toll-free, 1-866-906-6100 or e-mail email@example.com. For more information about
VIMIZIM, please visit www.VIMIZIM.com.
Clinical Trial Results
The safety and efficacy of VIMIZIM were assessed in a 24-week, randomized,
double-blind, placebo-controlled clinical trial of 176patients with MPS IVA
(MOR-004). The primary endpoint of the trial, change in six-minute walk
distance at 24 weeks, was statistically significant in patients receiving
weekly infusions of VIMIZIM at the dose of 2 mg/kg with a mean increase of
22.5 meters (p=0.0174) over placebo.
In patients who continued to receive VIMIZIM 2 mg/kg once per week for another
48weeks (for a total of 72-week exposure), walking ability was sustained to a
similar level that was achievedduring the first 24 weeks of treatment in the
placebo-controlled trial, MOR-004.
Overall, sustained improvements across multiple efficacy measurements and
across multiple clinical trials provided evidence of clinical benefit to
patients with MPS IVA, a chronic, progressive disease in which clinical
deterioration is the expected course.
The adverse events observed in clinical trials were similar to those seen in
other enzyme replacement therapies. In the Phase 3 trial, the most common
adverse reactions (≥10% and a higher incidence than placebo) that occurred
were pyrexia, vomiting, headache, nausea, abdominal pain, chills, and
fatigue. No new types of adverse reactions were reported in the Phase 3
extension trial. The most common adverse reactions (≥10%) observed across
pre-marketing clinical trials were similar in type and frequency as those
observed in the placebo-controlled trial. Acute reactions requiring
intervention were managed by either temporarily interrupting or discontinuing
infusion, and administering additional antihistamine, antipyretics, or
Note to Investors
BioMarin will host a webcast to discuss the VIMIZIM approval Tuesday, February
18, 2014 at 5:00 a.m. PT. Dial-in information for the conference call will be
distributed prior to the call.
Interested parties may access a live audio webcast of the conference call via
the investor section of the BioMarin website, www.BMRN.com.A replay of the
call will be archived on the site for one week following the call.
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.
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 from 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
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.
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, 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) 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 in countries
other than the United States, 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 and predictions
and involve risks and uncertainties such that actual results may differ
materially from these statements.These risks and uncertainties include, among
others: actions by regulatory agencies other than the FDA, results and timing
of current and planned clinical trials of its products; the availability and
launch of VIMIZIM in the United States 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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