Not Such A Sure Thing: FDA Knocks Back BioMarin’s Roctavian
Company’s Hemophilia A Gene Therapy Gets A CRL
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.
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Join us for a quick audio roundup of five useful things to know from Scrip's recent coverage of the global biopharmaceutical sector.
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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.