BioMarin scores a quick FDA OK for its latest rare disease entry — flagged at $702K WAC
The FDA has provided a widely expected green light for BioMarin’s Brineura (cerliponase alfa), setting up the rollout for one of the most expensive new drugs to hit the market.
BioMarin $BMRN attracted attention for its latest rare disease drug for its efficacy in treating Neuronal Ceroid Lipofuscinosis Type 2 disease (CLN2), a form of Batten disease. The disease progresses rapidly, with recurrent seizures and vision loss occurring as most victims are quickly left unable to walk by the age of six before eventually killing them. And just days ago the European Medicines Agency’s Committee for Medicinal Products for Human Use provided their thumbs up as well.
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