Orchard’s MLD gene therapy becomes costliest US medicine

rare disease MLD
Марина Вельможко/marvelmozhko

Orchard Therapeutics has revealed its US pricing plans for Lenmeldy, its gene therapy for metachromatic leukodystrophy (MLD), placing a $4.25 million price tag on the one-shot treatment.

On Monday, Lenmeldy (atidarsagene autotemcel) became the first FDA-approved treatment for children with MLD, a rare genetic disorder that causes rapid deterioration in motor and cognitive function. The life expectancy for infants born with the disease is typically five years or less, while those with later-onset forms generally don’t live beyond 20.

Orchard, which was recently acquired by Kyowa Kirin, said in a statement that the wholesale price of the gene therapy – the highest for any medicine in the US – reflects its “clinical, economic, and societal value,” as Lenmeldy can stop or slow the progression of MLD, potentially allowing some children to live a normal life.

The wholesale price is a little higher than the $3.7 million Orchard charges for the drug in Europe, where it has been launched in several markets as Libmeldy, and is also above the $2.3 million to $3.9 million range that the Institute for Clinical and Economic Review (ICER) said would be cost-effective in a report published last year.

Orchard’s chief operating officer, Frank Thomas, said the company is “confident in the potential long-term clinical outcomes of Lenmeldy.”

He added efforts are underway to explore “structure outcomes-based and other types of innovative reimbursement models that appropriately balance the needs of patients and families for adequate access, health care systems for affordability, as well as support future research and development of treatments for ultra-rare diseases like MLD.”

In a trial of 37 children, the treatment allowed all 37 to survive to the age of six, compared to just 58% of the group in historical controls. 71% of treated children could walk without assistance and 85% had normal language and performance IQ scores.

Five specialised centres for delivering Lenmeldy treatment are in the process of being qualified across the US, which the company said would minimise the travel burden on eligible patients and their families. Orchard is also offering a personalised support programme for patients called Orchard Assist, as well as “outcomes- and value-based agreements” with both private and government payers.

Fewer than 40 children are diagnosed with MLD each year in the US, but the lengthening list of highly-priced gene therapies for rare diseases has raised concerns that, collectively, the treatments may become difficult for healthcare systems to afford.

A study published in the journal Nature last year suggested that the annual spending on gene therapies in the US could reach approximately $20.4 billion at conservative estimates, excluding the costs of delivering therapy such as in-hospital care. According to that model, Medicare would face a bill of around $5.4 billion, which is less than 1% of its 2018 spending of $597.4 billion.

Image by Марина Вельможко from Pixabay