Rare genetic disease treatment becomes the world’s most costly drug at $4.25mn

In the US, a new gene therapy that treats a rare genetic disorder that attacks the nervous system central of young children is priced at $4.25mn. This makes it the most costly drug in history.

Lenmeldy, a medicine developed by Orchard Therapeutics, became the first US-approved treatment for metachromatic lukodystrophy this week. MLD is a genetic disorder that affects 40 babies in the US on average every year.

In the most severe case of MLD, children who are in their late infancy lose all motor functions and eventually become vegetative. They require round-the clock intensive care. The majority of patients die five years after symptoms begin.

Orchard, the UK-based biotech recently acquired by the Japanese pharmaceutical company Kyowa Kirin argued that there were no other treatments available for MLD, which justified the high price. The decision has reignited the debate over the price of cell and gene therapy, which offers new and revolutionary treatments and can even cure patients who have very few options.

The most expensive gene therapies approved are uniQure’s Hemgenix for haemophilia A, priced at $3.5mn, and Bluebird’s Zynteglo for thalassaemia, priced at $2.8mn.

Bobby Gaspar is the co-founder and CEO of Orchard. He said Lenmeldy “is a paradigm-shifting drug”. The company “is committed to enabling wide, expeditious and sustainable access for eligible patients in the US with early-onset MLD”.

Orchard’s MLD treatments was listed at a price of $3.9 million when it was approved in Europe under the brand name Libmeldy in 2020, but national healthcare systems negotiated steep discounts. It was €2.4mn in Germany.

Orchard cited a study by the Institute for Clinical and Economic Review, a US non profit that attempts to determine a fair price for medications. The review concluded that Lenmeldy was cost-effective if it were priced between $2.3mn and $3.9mn.

Gaspar said that Orchard “always took on board” ICER’s evaluation, but had decided to increase the price of the drug. He said: “That is our list price. We will enter into discussions with payors and there will be confidentiality discounts.”

Gaspar said that without Lenmeldy “these children would all die in their first or second decade”. Gaspar said, “Up until the medicine was available, there was no way to stop this relentless decline.”

Orchard reported that the drug had generated revenues of $12.7mn over the nine-month period ending in September.

Bennett Smith, Orchard’s senior vice president and general manager for North America, said: “MLD is a devastating disease that places a heavy emotional and financial burden on families. They face significant wage losses and additional expenses every year as the illness progresses. All this while coping with the incomprehensible pain of losing a child.”

Lenmeldy is a drug that works by injecting a genetically-modified version of the gene responsible for MLD into a patient’s stem cells. It then delivers it through an infusion. In a study of 37 children with early-onset MLD who were treated with Lenmeldy at six years old, all the children survived, while only 58% in the control group did. At five years old, 71% of patients were able to walk unaided and 85% had normal scores in language and cognition.