BioMarin's Brineura Approval Shows FDA's Open Door For Orphan Drugs
Executive Summary
Filing supporting Brineura was supported by a tiny, single-arm study and had no advisory committee review, the latest sign of the US regulatory agency's willingness to speed orphan drugs for serious pediatric diseases.
You may also be interested in...
Amicus Makes Its Gene Therapy Move, Focusing On CNS Lysosomal Storage Disorders
The rare disease specialist gained 10 gene therapy programs with the acquisition of Celenex, including two in clinical-stage development for Batten disease, in exchange for $100m up front. President Bradley Campbell talked to Scrip about the process that led to the deal and next steps for development.
A Baker’s Dozen Of US FDA Efficacy Approvals Using Real World Evidence
Fresenius Kabi’s Omegaven is most recent of 13 approvals, including three with breakthrough therapy designations, where FDA has relied on real world evidence to make a regulatory decision about efficacy.
Priority Review Voucher Sales Prices Level Off In 2017
BioMarin's sale of voucher earned with approval of rare pediatric disease drug Brineura the fourth publicly disclosed voucher sale this year with a price tag between $125m and $150m.