-- Scott Roberts
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
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
The FDA has more about
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