Orchard’s Lenmeldy won FDA approval, with the price tag of $4.25 million, the therapy quickly took the title of most expensive drug

FDAs Peter Marks, M.D., Ph.D., receives a simple email from Commissioner Robert Califf, M.D. each time a gene therapy gets approved.

What the Hell, it’s Only $4.25 million for a single patient!

Fierce Pharma spreads the ‘exciting’ news once again:

For every million-dollar gene therapy, Peter Marks gets an email from FDA chief Robert Califf

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)…

…in March, Orchard’s Lenmeldy won FDA approval to treat the rare genetic disease metachromatic leukodystrophy (MLD). With the price tag of $4.25 million, the therapy quickly took the title of most expensive drug, taking a crown previously w…