Orchard Therapeutics made history this week when the U.S. Food and Drug Administration approved its gene therapy as the first treatment for children with a rare, deadly genetic disease called metachromatic leukodystrophy (MLD).
Now, the biotech, which is based in London but has it's U.S. headquarters in Boston's Seaport neighborhood, has made history again, pricing its one-time treatment at $4.25 million. That makes it the most expensive drug in history.
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On Wednesday, Orchard (Nasdaq: ORTX) announced the wholesale acquisition cost of its treatment, which will be marketed as Lenmeldy in the U.S. It’s a record-setting price for a drug to treat a rare and fatal disease for which there has previously been no treatment options beyond supportive and end-of-life care.
Orchard, which was recently bought by Japanese pharmaceutical giant Kyowa Kirin Co. Ltd., said MLD affects fewer than 40 children annually in the U.S.
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