FDA Approves Vimizim (Elosulfase Alfa) to Treat Rare Congenital Enzyme Disorder
First drug to receive rare pediatric disease priority review voucher (February 14)
The FDA has approved Vimizim (elosulfase alfa, BioMarin Pharmaceutical Inc.), the first agency-approved treatment for mucopolysaccharidosis type IVA (Morquio A syndrome). 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. Absence of this enzyme leads to problems with bone development, growth, and mobility. There are approximately 800 patients with Morquio A syndrome in the U.S.
Vimizim was granted priority review. It was also the first drug to receive the Rare Pediatric Disease Priority Review Voucher — a provision that aims to encourage the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.
The safety and effectiveness of Vimizim were established in a clinical trial involving 176 participants with Morquio A syndrome ranging in age from 5 to 57 years. Participants treated with Vimizim showed greater improvement in a 6-minute walk test than did participants given placebo. On average, patients treated with Vimizim walked 22.5 meters farther in 6 minutes compared with the patients who received placebo.
The most common side effects in patients treated with Vimizim during clinical trials included fever, vomiting, headache, nausea, abdominal pain, chills, and fatigue. 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 that includes the risk of anaphylaxis. During clinical trials, life-threatening anaphylactic reactions occurred in some patients during Vimizim infusions.
Source: FDA; February 14, 2014.