BioMarin’s Vimizim Secures Broad Label In Morquio A Syndrome
This article was originally published in The Pink Sheet Daily
No limits on age or disease severity of patients appear in elosulfase alfa labeling; agency reviewers had raised the issue of baseline function at a November advisory committee meeting.
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Vimizim post-marketing requirements could help FDA better understand the clinical significance of anti-drug antibodies commonly seen with enzyme replacement therapies for lysosomal storage diseases and the risks of tolerance induction therapy to manage immunogenicity.
FDA clearance of the Morquio A syndrome treatment provides some insight into how the agency is implementing the 2012 priority review voucher program aimed at incentivizing development of treatments for rare pediatric diseases.
Europe’s top scientific advisory panel also gave positive recommendations to BioMarin’s orphan drug Vimizim for use in mucopolysaccharidosis type IVA, and the compassionate use of a fixed combination of two of Gilead Science’s antivirals in hepatitis C, sofosbuvir and ledipasvir, after its meeting Feb. 17-20.