NICE Recommends BioMarin's Vimizim® (elosulfase alfa) for the Treatment of Morquio A Syndrome in England

Monday, 23. November 2015 16:59

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.

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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

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.

Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558

Judith Moore
On behalf of BioMarin Pharmaceutical Inc.
+44 (0)20 7632 1806

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Source: BioMarin Pharmaceutical Inc. via Globenewswire

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