SAN RAFAEL, Calif.,
June 4, 2019 /PRNewswire/
-- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today
that Vimizim® (elosulfase alfa) has been approved by the National
Medical Products Administration (NMPA) for the treatment of
patients with mucopolysaccharidosis type IVA (MPS IVA), also known
as Morquio A syndrome. Vimizim is the first treatment in
China approved for this
condition.
In May 2018, the Chinese
government issued the country's first national list of rare
diseases, which included MPS. The list also includes
phenylketonuria (PKU), Tetrahydrobiopterin Deficiency, and
hemophilia—diseases that BioMarin either has an approved therapy
for or is developing one. The list gives priority to rare
diseases with a relatively high prevalence, that pose a heavy
burden and that are highly treatable. Also, in August 2018, the Chinese Drug Evaluation Center
posted a list of 48 drugs already approved in the U.S., EU or
Japan that could be eligible for
Priority Review in China, which
included Vimizim.
"Vimizim is the first, and currently only, disease-specific
treatment option for this very rare, progressively degenerative,
autosomal-recessive lysosomal storage disorder," said Luo Xiaoping,
Vice Chairman of the Pediatrics society of the Chinese Medical
Association and head of Endocrine Genetic Metabolism Group of
Chinese Medical Association.
"Morquio A syndrome is an ultra-rare and difficult condition to
treat. Vimizim is the only specific treatment available and offers
improved endurance to these patients," said Gu Xuefan, Deputy
Director of Shanghai Institute of Pediatrics, Director of Pediatric
Endocrinology and Inherited Metabolic Diseases Research Office of
Xinhua Hospital, Director of Pediatric Genetic Diseases Diagnosis
and Treatment Center, and Director of Pediatric Endocrinology and
Inherited Metabolism Group of Shanghai Medical Association.
"Vimizim is approved for patients with mucupolysaccharidosis
type IVA. It's used to improve the patients' pulmonary function,
and significantly increase their walking distance in six minutes,"
said Qiu Zhengqing, Member of the Pediatric Endocrinology and
Metabolism Group of the Chinese Medical Association, communication
reviewer of Chinese Journal of Pediatrics and the Chinese Medical
Genetics Journal.
"We are pleased to be able to deliver the first drug therapy for
Morquio A to patients in China and
to build upon our efforts to serve patients with rare diseases in
China," said Jean-Jacques
Bienaimé, Chairman and CEO of BioMarin. "We hope to continue to
deliver therapies to treat patients who have rare genetic diseases
with unmet medical needs."
BioMarin will market Vimizim directly in China, building on its existing presence in
the country with a team that has been marketing a BioMarin therapy,
Kuvan® (sapropterin dihydrochloride), and that team is well-suited
for serving ultra-rare disease populations. In the U.S., Kuvan is
approved to reduce blood phenylalanine (Phe) levels in patients
with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-)
responsive Phenylketonuria (PKU).
Morquio A syndrome is an ultra-rare, severely debilitating
disease affecting 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.
"The approval of Vimizim in China underscores our commitment to providing
this much-needed therapy to patients with Morquio A syndrome across
the globe," said Hank Fuchs, M.D.,
President of Global Research and Development at BioMarin. "We will
continue to seek approvals in other countries so that more patients
within the MPS community have access to the treatments they
deserve."
About Morquio A Syndrome
Morquio A syndrome, or Mucopolysaccharidosis IVA (MPS IVA) is a
disease in which people are missing an enzyme 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,
limiting mobility and endurance. Malformation of the chest impairs
respiratory function, and looseness of joints in the neck causing
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.
About VIMIZIM
VIMIZIM® (elosulfase alfa) is a treatment for patients with
Morquio A syndrome, or mucopolysaccharidosis IVA (MPS
IVA). VIMIZIM is the first approved 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
approved, standard-of-care treatment other than supportive
care.
Important Safety Information from U.S. Prescribing
Information
Life-threatening allergic reactions, known as anaphylaxis, can
occur during VIMIZIM® (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
clinical care.
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 been established.
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 www.VIMIZIM.com.
About BioMarin
BioMarin is a global biotechnology company that develops and
commercializes innovative therapies for serious and
life-threatening rare and ultra-rare genetic diseases. The
Company's portfolio consists of seven commercialized products and
multiple clinical and pre-clinical product candidates. For
additional information, please visit www.biomarin.com.
Information on BioMarin's website is not incorporated by reference
into this press release.
Forward Looking Statements
This press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc., including,
without limitation, statements about: expectations regarding the
marketing and commercialization of Vimizim in China. 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;
any further actions by the NMPA; the outcome of pricing and
reimbursement negotiations with relevant authorities in
China; 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" and elsewhere in BioMarin's Securities and
Exchange Commission (SEC) filings, including the Current Report on
Form 10-Q for the quarter ended March 31,
2019, and future filings and reports by BioMarin. BioMarin
undertakes no duty or obligation to update any forward-looking
statements contained in this press release as a result of new
information, future events or changes in its expectations.
BioMarin® Vimizim®, and Kuvan® are registered trademarks of
BioMarin Pharmaceutical Inc.
Contacts:
|
|
Investors
|
Media
|
Traci
McCarty
|
Debra
Charlesworth
|
BioMarin
Pharmaceutical Inc.
|
BioMarin
Pharmaceutical Inc.
|
(415)
455-7558
|
(415)
455-7451
|
View original content to download
multimedia:http://www.prnewswire.com/news-releases/biomarin-announces-approval-of-vimizim-elosulfase-alfa-in-china-for-treatment-of-morquio-a-syndrome-300861353.html
SOURCE BioMarin Pharmaceutical Inc.