FDA approves Vimizim for MPS IV A - BioMarin

The FDA has approved Vimizim (elosulfase alfa), from BioMarin, for patients with Mucopolysaccharidosis type IVA (MPS IV A or Morquio A syndrome). Approval is based on Phase III trials, in which Vimizim was shown to significantly improve endurance, which possibly could change the course of the disease.

The primary endpoint of the trial, a change in six-minute walk distance at 24 weeks, was statistically significant in patients receiving weekly infusions of Vimizim at the dose of 2 mg/kg with a mean increase of 22.5 meters (p=0.0174) over placebo. In patients who continued to receive Vimizim 2 mg/kg once per week for another 48 weeks (for a total of 72-week exposure), walking ability was sustained to a similar level that was achieved during the first 24 weeks of treatment in the placebo-controlled trial, MOR-004. Overall, sustained improvements across multiple efficacy measurements and across multiple clinical trials provided evidence of clinical benefit to patients with MPS IVA, a chronic, progressive disease in which clinical deterioration is the expected course. The adverse events observed in clinical trials were similar to those seen in other enzyme replacement therapies.