mohd izzuan
Orchard Therapeutics (ORTX), recently acquired by Kyowa Kirin (OTCPK:KYKOY), has set its newly FDA-approved rare disease therapy Lenmeldy at a wholesale price of $4.25M on Wednesday, making one-time gene therapy the world's costliest medicine.
Early this week, the FDA approved Lenmeldy as the first U.S. treatment for early-onset metachromatic leukodystrophy (MLD), a rare metabolic disorder.
The British company noted that the drug's wholesale acquisition cost (WAC) which indicates the drug's list price, reflects the value its medicine delivers to patients, their families, and the overall healthcare system.
WAC excludes elements such as rebates and discounts and may not be a patient's final cost, which also depends on health insurance.
The company added that its pricing was in line with a recent review by the drug pricing watchdog, the Institute for Clinical and Economic Review (ICER). The non-profit indicated up to a $3.94M health benefit price benchmark for Lenmeldy.
Previously, hemophilia B therapy, Hemgenix, was the world's most expensive drug when its developers, Australia-based CSL (OTCQX:CSLLY) (OTCQX:CMXHF) and German biotech uniQure (QURE), launched the one-time gene therapy at $3.5M.
Sarepta Therapeutics (SRPT) was close behind with its $3.2M price tag for its recently approved Duchenne muscular dystrophy (DMD) therapy. Elevidys, just ahead of bluebird bio's (BLUE) Skysona. The one-time gene therapy was launched at $3.0M for a rare neurodegenerative disease called cerebral adrenoleukodystrophy in late 2022.