Speedy review and no adcomm for BioMarin's pioneering hemophilia gene therapy
BioMarin’s keenly anticipated hemophilia A gene therapy — which CEO JJ Bienaimé envisions pricing at an eye-watering $2 million to $3 million a shot — has secured red carpet treatment at the FDA.
The therapy, fondly called valrox, has won priority review — and there the agency has indicated there are no plans for an advisory committee meeting. The US regulator’s final decision is expected by August 21. If approved, it will be the pioneering gene therapy for hemophilia.
“While the company…indicated that FDA does not currently plan on convening an advisory committee meeting to discuss the merits of the application, we caution that the possibility of such still remains as the agency digs into its review, which could be interpreted negatively by the Street,” BMO Capital Market’s George Farmer wrote.
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