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Vimizim Approved for Rare Childhood Disorder

Vimizim Approved for Rare Childhood Disorder


MONDAY, Feb. 17, 2014 (HealthDay News) -- Vimizim (elosulfase alfa) has been approved by the U.S. Food and Drug Administration to treat a rare childhood disorder called Mucopolysaccharidosis Type IVA, also known as Morquio A syndrome.

The disorder is caused by a missing metabolic enzyme that leads to problems with bone development, growth and movement, the agency said in a news release. It affects about 800 people in the United States.

Vimizim replaces the missing enzyme, known as GALNS. The drug's safety and effectiveness were established in clinical trials involving 176 people, ranging in age from 5 to 57. The most common side effects included fever, vomiting, headache, nausea, abdominal pain, chills and fatigue, the FDA said.

The drug's safety and effectiveness weren't evaluated in children under age 5 years, the agency added. Vimizim's label will include a boxed warning to include the risk of anaphylaxis, an allergic-like reaction that could be life threatening.

Vimizim was the first drug granted a Rare Pediatric Disease Priority Review Voucher, an FDA effort to encourage development of new treatments for rare childhood diseases.

The drug is marketed by BioMarin Pharmaceuticals, based in Novoto, Calif.

More information

The FDA has more about this approval.

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Please be aware that this information is provided to supplement the care provided by your physician. It is neither intended nor implied to be a substitute for professional medical advice. CALL YOUR HEALTHCARE PROVIDER IMMEDIATELY IF YOU THINK YOU MAY HAVE A MEDICAL EMERGENCY. Always seek the advice of your physician or other qualified health provider prior to starting any new treatment or with any questions you may have regarding a medical condition.


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