The world has appeared a new The most expensive drug is gene therapy worth $4.25 million. The medication, called Lenmeldy, is designed to treat metachromatic leukodystrophy (MLD) – a rare genetic disease that affects children and rapidly robs them of the ability to speak and walk. Approximately half of the affected children die, while the rest live in a near-vegetative state, placing a heavy burden on their families.
The drug has been approved in the United States, and its manufacturer, Orchard Therapeutics, asserts that the price reflects the therapy’s value for patients and their families. Despite its high cost, the commercial success of Lenmeldy is not guaranteed, as genetic therapies have faced market challenges in the past.
The efficacy of Lenmeldy, which has been undergoing testing since 2010, is unquestionable: the therapy restores the missing gene in children’s bone marrow cells, addressing the root cause of the brain disease. Children who undergo treatment are able to resume normal life, walk, and engage in sports.
However, the high prices of such drugs raise concerns about their economic sustainability. Previously, another genetic therapy, Glybera, was withdrawn from the market due to inadequate effectiveness and high costs. Orchard has also faced a similar issue, discontinuing the genetic therapy Strimvelis due to limited patient numbers and alternative treatment options.
In the case of Lenmeldy, early disease diagnosis will be crucial, as symptoms may appear too late for effective treatment. The company aims to have the disease included in routine newborn screening, which could expand the drug market and save more children.
Among the supporters of the therapy is Amy Price, an advocate for patients with rare diseases who lost one child to MLD but was able to save two others through genetic therapy. According to her, the economic burden of an affected child outweighs any cost of gene therapy, highlighting the challenge of understanding reality when people focus solely on pricing.