Recommendation Provides Patient Access to Treatment for Ultra-Rare
Condition With Reimbursement
SAN RAFAEL, Calif., Nov. 23, 2015
(GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN)
today announced that 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. "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.
"This is fantastic news for all
affected patients," said Professor Chris Hendriksz of Salford Royal
NHS Foundation Trust. "Many of the patients who participated in the
largest ever clinical trial for ultra rare enzyme replacement
therapies are from England, and today's announcement means they and
all diagnosed patients in the country will now have a greater
opportunity to access treatment. The decision by NICE also
means many patients will have the possibility of access to
treatment at home, which will put an end to long journeys to
hospital to receive weekly treatment. The impact for patients will
be significant. Previously, we have only been able to help manage
symptoms, but this new treatment is now able to target the
underlying cause of this devastating disorder."
"As Vimizim is the first and only
treatment to address the underlying cause of Morquio A syndrome, we
applaud the NICE'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 NICE, 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 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
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 foetus. It is not known if Vimizim is present in human
milk.
Safety and effectiveness in
paediatric patients below 5 years of age have not been established.
In clinical trials, the most common adverse reactions (greater than
or equal to 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. The Summary of Product
Characteristics can be found
at: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/002779/WC500169239.pdf.
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 BioMarin
BioMarin is a global biotechnology
company that develops and commercialises innovative therapies for
patients with serious and life-threatening rare and ultra-rare
genetic diseases. The company's portfolio consists of five
commercialised products and multiple clinical and pre-clinical
product candidates. For additional information, please
visit www.BMRN.com.
Forward-Looking Statement
This 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.