FDA Approves Vimizim – New Drug For Murquio
On February 14, 2014 the U.S. Food and Drug Administration approved a new drug treatment for patients with mucopolysaccharidosis Type IVA, also known as Murquio A Syndrome. The new medication goes by the trade name Vimizim (chemical name elosulfase alfa), and it is marketed in the U.S. by Biomarin, Inc.
Murquio A Syndrome is a rare disease that affects approximately 800 individuals in the United States. Morquio A syndrome is a rare, autosomal recessive lysosomal storage disease caused by a deficiency in N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Vimizim is intended to replace the missing GALNS enzyme involved in an important metabolic pathway. Patients with Murquio A Syndrome have problems with bone development, growth and mobility. The FDA approved Vimizim under a Priority Review with an orphan drug status.
The most common side effects seen in patients treated with Vimizim during clinical trials included nausea, vomiting, abdominal pain, headache, fever, chills and fatigue. At this time, FDA-approved labeling states that the safety and effectiveness of Vimizim have not been established in pediatric patients less than 5 years of age. Vimizim is being approved with a boxed warning to include the risk of anaphylaxis.
Vimizim launch date and estimated Wholesaler Acquisition Cost (WAC) for Vimizim are not known at the time of this posting.
For more information, see the Vimizim information on the manufacturer’s website at Biomarin, Inc. or the FDA’s news release at: Link to FDA Vimizim news release