New advanced therapeutic medicines are reaching the market, but their price tags remain exceptionally high. This year’s ranking of the most expensive drugs in the US reveals a common denominator: all are gene therapies administered as one-time treatments.
The Japanese pharmaceutical company Kyowa Kirin acquired Orchard Therapeutics in the autumn of 2023, thereby gaining access to what is now the world’s most expensive drug – Libmeldy (marketed as Lenmeldy in the U.S.).
Last year, the company received FDA approval for this gene therapy, which is indicated for the treatment of the rare neurological disorder metachromatic leukodystrophy (MLD). The U.S. price has been set at $4.25 million per dose. The therapy had already been approved in the EU in 2020.