Fierce Pharma May 8, 2024
After Orchard Therapeutics’ gene therapy for a rare genetic disease was approved in March, Peter Marks, M.D., Ph.D., received a simple email from Commissioner Robert Califf, M.D.
“Every time we approve a new gene therapy, and then the company announces the price of the gene therapy, the commissioner sends me a note and it’s usually [short]—I love short emails,” Marks told the crowd at the American Society of Gene and Cell Therapy annual meeting Wednesday morning. Marks serves as director of the Center for Biologics Evaluation and Research (CBER).
“The last one was a $4.25 million exclamation point,” Marks recalled. “It was like a lot of exclamation points after. I can’t remember how many, and that was the email....
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