Medical bills have never looked higher. A gene therapy for a rare disease afflicting infants has set its price: $4.25 million, making it the most expensive drug in the world.
Pharma company Orchard Therapeutics, which develops treatments for severe and genetic diseases, set the wholesale cost for its new therapy Lenmeldy just days after it was approved by the U.S. Food and Drug Administration (FDA).
The London-based company said in a press release on Wednesday the high price was “reflective of the value the therapy may deliver to eligible patients and their families.” The company added that price also takes into account the long-term impact the treatment will have on overall healthcare spending.
Lenmeldy is a one-time, single dose infusion made of a patient’s own blood and stem cells to treat infants with early metachromatic leukodystrophy (MLD). The rare genetic disease results in the build up of fatty substances called sulfatides in the cells of the brain, nervous system, and other organs. It can result in loss of motor and cognitive function, and even death. The FDA said that MLD affects one in 40,000 Americans.
Orchard Therapeutics was acquired by the Japanese pharma company Kyowa Kirin in January (pdf) for about $477 million.
New treatments for rare diseases have boosted drug prices
The previous title holder for world’s most expensive drug was CSL Behring’s Hemgenix. The gene therapy, which launched in 2022, costs $3.5 million and is used to treat the bleeding disorder hemophilia B.
Expensive treatments like Lenmeldy and Hemgenix boosted average prices for new drugs last year.
Pharmaceutical companies set prices for new drugs in the U.S. 35% higher in 2023 than they did the prior year, according to a new analysis by Reuters.
The steep price hike comes as the industry is embracing treatments for rare disease, also called orphan diseases. These illnesses typically affect a small population, usually 200,000 people or less. Drug companies are able to leverage a lack of alternative treatments for these diseases as justification for high prices.
The median annual list price for a new medication in 2023 was $300,000, up 35% from $222,000 in 2022. The median list price for about 30 new drugs in 2021 was $180,000.