CHMP Clears Pierre Fabre’s Hemangiol For Children, Under Rarely Used Pediatric Pathway
This article was originally published in The Pink Sheet Daily
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.
You may also be interested in...
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.
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.
The European pharmaceutical industry has refuted claims that manufacturers are exploiting a loophole in EU legislation to avoid running non-lucrative trials for pediatric oncology drugs.