BioMarin Could Launch Brineura Concurrently In US And Europe
The EU’s top advisory panel, the CHMP, has given a green light to BioMarin’s late-infantile Batten disease therapy, Brineura, one of the first therapies for the ultra-rare and fatal disease, a decision that comes just days before a US FDA decision is expected on the replacement enzyme therapy.
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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.
Keeping Track: Breakthroughs Dominate 2017 Novel Agents; US FDA Approves Rydapt, Alunbrig, Tymlos, Brineura
The latest drug development news and highlights from our FDA Performance Tracker.
Fast US approval of ultra-rare pediatric disease drug Brineura (cerliponase alfa), with an annual list price of $702,000, is a victory for the company, but approval for restricted pediatric population compared to Europe raised questions.