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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The company is aiming for an outcomes-based payment model in the US, while it is confident the gene therapy will fare better than some others have in Europe.
Very early launch numbers look good in France and Germany, but US launch will be more challenging due to a fragmented patient base, the company said. Launch price is a flat $899 per vial.
A patient developed Factor VIII inhibitors, but he had also recently received a COVID-19 vaccine and was genetically predisposed.