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FDA Panel Backs Approval of Vimizim for Treatment of Rare Genetic Disorder

Committee cites positive phase III data (November 19)

The FDA’s Endocrine and Metabolic Drugs Advisory Committee has recommended the approval of an experimental drug, elosulfase alfa (Vimizim, BioMarin Pharmaceutical Inc.), to treat Morquio A syndrome, a rare genetic disorder that causes skeletal malformation and a variety of related lung, eye, ear, and heart problems.

The opinion was issued to the public prior to a formal meeting of the committee on November 19. In a pivotal phase III trial, Vimizim, at a dosage of 2.0 mg/kg per week, demonstrated a statistically significant improvement at week 24 in the study’s primary efficacy measure, the 6-minute walk test distance, compared with placebo. The treatment effect was 22.5 meters (P = 0.0174) for the active treatment. None of the patients stopped treatment because of an adverse event.

The advisory panel concluded that the safety and efficacy data derived from the clinical development program for Vimizim demonstrated clinical benefit and acceptable tolerability of the drug at the proposed marketed dose of 2.0 mg/kg per week, and support “an overall positive risk-benefit profile in the target patient population.”

The FDA is not obligated to follow the recommendations of its advisory panels but usually does so.

[Source: FDA; November 19, 2013.]

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