Scrip is part of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By

UsernamePublicRestriction
UsernamePublicRestriction

BioMarin Expects 30% Markdown For Orphan Pediatric Drug Brineura

Executive Summary

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.

You may also be interested in...



BioMarin's Brineura Approval Shows FDA's Open Door For Orphan Drugs

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.

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.

Related Content

Topics

Related Companies

UsernamePublicRestriction

Register

LL1134478

Ask The Analyst

Ask the Analyst is free for subscribers.  Submit your question and one of our analysts will be in touch.

Your question has been successfully sent to the email address below and we will get back as soon as possible. my@email.address.

All fields are required.

Please make sure all fields are completed.

Please make sure you have filled out all fields

Please make sure you have filled out all fields

Please enter a valid e-mail address

Please enter a valid Phone Number

Ask your question to our analysts

Cancel