BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that the
The National Institute for Health and Care Excellence (NICE), NHS
England and BioMarin have reached an agreement on a Managed Access
Agreement, which provides a basis for access for clinically
suitable mucopolysaccharidosis type IVA (MPS IVA) patients to
Vimizim® (elosulfase alfa) treatment for the next five years.
The provision of access is subject to the completion of the ongoing
NICE Highly Specialised Technologies process, where the final
guidance is expected in mid-December.
“The Morquio A community has been long awaiting this decision,
which provides patients access to Vimizim, and we are relieved and
elated to have resolution,” said Christine Lavery, Chief Executive
of the MPS Society, (United Kingdom). “With fewer than 80 people
living with Morquio A syndrome in England, it is hard to comprehend
what these patients and their families have been through over the
last 18 months. Treatment to this community is so much more than
just a therapy. It is hope for a future where their improved
health allows them to reach their full potential.”
Morquio A syndrome is an ultra-rare, severely debilitating
disease affecting an estimated 3,000 patients in the developed
world. 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.
“As Vimizim is the first and only treatment to address the
underlying cause of Morquio A syndrome, we applaud the Institute’s
decision to provide the patient community with access to this
much-needed therapy,” said Jim Lennertz, Group Vice President and
Regional Manager of Europe, the Middle East and Africa at BioMarin.
“As part of our commitment to the MPS community, we worked
diligently with the Institute, advocates and patients to bring
Vimizim to those who need it.”
The U.S. Food and Drug Administration (FDA) approved the Vimizim
license application for the treatment of patients with Morquio A
syndrome on February 14, 2014, and the European Commission approved
it on April 28, 2014. The therapy is also approved in
Australia, Canada, Brazil and Japan.
About VimizimVimizim®
(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
InformationLife-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 Morquio A SyndromeMorquio
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 BioMarinBioMarin is a global
biotechnology company that develops and commercializes innovative
therapies for patients with serious and life-threatening rare and
ultra-rare genetic diseases. The company's portfolio consists of
five commercialized products and multiple clinical and pre-clinical
product candidates. For additional information, please visit
www.BMRN.com.
Forward-Looking StatementThis
press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc., including,
without limitation, statements about: expectations regarding NICE,
NHS England and BioMarin reaching an agreement on a Managed Access
Agreement for reimbursement for Vimizim. 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 provision of access is subject to the completion of the
ongoing NICE Highly Specialised Technologies process, actual
reimbursement decisions by NHS England; the acceptability of final
terms of the Managed Access Agreement; 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® and Vimizim® are registered trademarks
of BioMarin Pharmaceutical Inc.
Investors:
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558
Media:
Debra Charlesworth
BioMarin Pharmaceutical Inc.
(415) 455-7451
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