FDA approves Vimizim for Morquio A syndrome

The FDA recently approved elosulfase alfa, an enzyme replacement treatment for patients with mucopolysaccharidosis type IVA, or Morquio A syndrome. According to a press release from the manufacturer, it is being approved with a boxed warning to include the risk for anaphylaxis.

Elosulfase alfa (Vimizim, BioMarin Pharmaceutical) is the first FDA-approved enzyme replacement therapy to target the underlying causes of the rare disease, which affects approximately 3,000 patients in the developed world. It also is the first agent to receive the rare pediatric disease priority review voucher. Morquio A syndrome, a type of lysosomal storage disorder, affects bone development, growth and mobility.

“This approval and rare pediatric disease priority review voucher underscores the agency’s commitment to making treatments available to patients with rare diseases,” Andrew E. Mulberg, MD, deputy director of the division of gastroenterology and inborn errors products in the FDA’s Center for Drug Evaluation and Research, said in the press release. “Prior to today’s approval, patients with this rare disease have had no approved drug treatment options.”

In December, the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted 19-2 to recommend its approval.

In clinical trials, patients administered elosulfase alfa demonstrated vast improvements in a 6-minute walk test. Patients treated with the drug walked 22.5 m farther in 6 minutes compared with patients administered placebo, according to the press release.

The most common adverse effects in patients treated with elosulfase alfa during clinical trials included fever, vomiting, headache, nausea, abdominal pain, chills and fatigue.