$4.25 million for the latest most expensive drug in the world
There’s a new most expensive drug ever – a gene therapy that costs as much as a house in Brooklyn or a mansion in Miami and more than the average person will earn in a lifetime.
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The therapeutic drug is called Lenmeldy and was developed for the treatment of metachromatic leukodystrophy (MLD). The drug received approval in the USA on Monday. Manufacturer Orchard Therapeutics says the $4.25 million cost reflects the value of the treatment to patients and families.
Small children are affected by the neurological disease MLD. The young patients lose the ability to speak and walk. About half die, the others continue to live in a vegetative state, which is unbearable for the families.
MLD is a rare disease: only about 40 children are diagnosed with it per year in the USA. Drugs that target these diseases are called orphan drugs. The extreme rarity of such diseases is the reason for the skyrocketing prices for new gene therapies. Just consider the economics: Orchard employs 160 people, far more than the number of children they can treat over several years.
Market opportunities for expensive gene therapies
As a result, even at that price, selling the latest DNA treatment could be a shaky business. “Gene therapies have struggled in the market – and I wouldn’t expect Lenmeldy to buck that trend,” says Maxx Chatsko, founder of Solt DB, which collects data on biotech products. If you will, being the most expensive drug in the world is a curse.
The MLD therapy was approved in Europe in 2021 under the name “Libmeldy”. The price is slightly lower at 2.8 million euros. But Chatsko points out that for most of last year, Orchard generated just $12.7 million from product sales. Accordingly, you can count the number of children who received it on your hands.
Gene therapy adds a missing gene to children’s bone marrow cells, eliminating the cause of the disease in the brain. Many of the children treated in the trials, which began in 2010, have developed into normal people.
The children can usually walk again and are cognitively fit. “The children we treat go to school, play sports and have their stories to tell,” says Leslie Meltzer, Orchard’s chief medical officer. Without the treatment this would not be possible.
Independent groups also believe the drug could be cost-effective. One of them, the Institute for Clinical and Economic Review, which evaluates the value of drugs, said last September that its models suggested MLD gene therapy would be worth it at a cost of between $2.3 million and $3.9 million. However, there is no denying that excessive prices can be a sign that a treatment is not economically viable.
Previous record holders
A previous title holder for the most expensive drug, the gene therapy Glybera, was bought just once before it was withdrawn from the market. It didn’t look good enough to justify the $1 million price tag that made it the price leader at the time.
The Novartis company launched the gene therapy Zolgensma for 2.125 million, for which it received FDA approval in 2019. It has also been on the market in Germany since mid-2020. It is a treatment for fatal muscle weakness in children (spinal muscular atrophy, type 1).
Hemgenix – also a gene therapy – replaced Zolgensma. It cost $3.5 million and was aimed at treating the genetic blood clotting disorder hemophilia B. Such treatments were expected to generate billions in sales, but according to press reports they are not being received nearly as well as one would expect.
Orchard itself has abandoned another DNA treatment, Strimvelis, costing just under €600,000, which is a full-fledged cure for a type of immune deficiency. The company owned the gene therapy and even approved it in Europe in 2016. The problem was both too few patients and the availability of alternative treatment. Not even a money-back guarantee could save Strimvelis, so Orchard discontinued it in 2022. In 2023, the Japanese drug manufacturer Kyowa Kirin took over the Orchard company, to which it still belongs today.
So gene therapies, although successful in trials, may lose the game. In Lenmeldy’s case, it will be crucial to detect the disease early. Because once children show symptoms, it can already be too late. So far, many patients have only been discovered because an older sibling already has the hereditary disease.
Expansion of newborn screenings
Orchard hopes to solve this problem by adding MLD to the list of diseases that are automatically tested for at birth. That would secure the market and save many more children. A decision on testing, advocates say, could come after a meeting of the U.S. Government Committee on Newborn Screening in May.
Proponents of the treatment include Amy Price, a rare disease treatment advocate who runs her own consulting firm, Rarralel, in Denver. Price had three children with MLD – one died, but two were saved by MLD gene therapy, which they received starting in 2011, when the treatment was still in testing.
Price says her two treated children, now teenagers, “are completely normal, absolutely average.” And that’s worth the price, she says. “The economic burden of an untreated child exceeds all previous gene therapy prices,” she says. “This reality is difficult to understand when people only want to respond to price.”
(jl)
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