Not Such A Sure Thing: FDA Knocks Back BioMarin’s Roctavian
Company’s Hemophilia A Gene Therapy Gets A CRL
Executive Summary
BioMarin’s confidence that its first-ever hemophilia gene therapy would be cleared by the US FDA was shaken by a complete response letter that the company claims changed the requirements for approval.
You may also be interested in...
Quick Listen: Scrip’s Five Must-Know Things
Join us for a quick audio roundup of five useful things to know from Scrip's recent coverage of the global biopharmaceutical sector.
BioMarin Files Vosoritide For Achondroplasia, But Investors Still Upset About Gene Therapy Setback
Vosoritide, a once daily injection, could be the first drug available for achondroplasia, the most common form of dwarfism.
uniQure Hemophilia B Deal With CSL Leaves M&A Fans Miffed
Observers have been expecting uniQure to be sold off as a whole but the Netherlands-based firm has chosen to sell just one asset - EtranaDez - and focus on advancing its Huntington’s disease gene therapy.
Existing Subscriber?
Sign in to continue reading.
Need a specific report? 1000+ reports available Buy Reports
Need a specific report? 1000+ reports available Buy Reports