Orchard Therapeutics Sets $4.25M Price for Gene Therapy

Orchard Therapeutics has received FDA approval for gene therapy Lenmeldy for the treatment of children with early-onset MLD, a rare and life-threatening inherited disease of the body’s metabolic system caused by a genetic mutation. The one-time, potentially life-saving treatment will carry a list price of $4.25 million, making the treatment the most-expensive ever approved according to the Boston Globe. Orchard’s U.S. headquarters is located in Boston.

“The value of Lenmeldy has been recognized by several…authorities around the world, including in the U.S. by ICER [the independent, non-profit Institute for Clinical and Economic Review], which determined Lenmeldy to have the highest value-based price for any treatment it has evaluated to date,” said Bennett Smith, senior vice president and general manager of North America at Orchard Therapeutics.

MLD is an ultra-rare, rapidly progressive, irreversible and ultimately fatal neurometabolic disease that affects approximately one in 100,000 live births, which is estimated to be fewer than 40 children annually in the U.S. It is caused by a mutation in the gene responsible for encoding the enzyme arylsulfatase A (ARSA) leading to neurological damage and developmental regression. Prior to Lenmeldy, there were no treatment options in the U.S. for early-onset MLD beyond supportive and end-of-life care.

Lenmeldy aims to correct the underlying genetic cause of MLD by inserting one or more functional copies of the human ARSA gene ex vivo (outside the body) into the genome of a patient’s own hematopoietic stem cells. The genetically repaired cells are infused back into the patient, where, once engrafted, they differentiate into multiple cell types, some of which migrate across the blood-brain barrier into the central nervous system and express the functional enzyme. This approach has the potential to restore enzymatic function to stop or slow disease progression with a single treatment.

Lenmeldy was granted Priority Review in September 2023. It was previously given both Rare Pediatric Disease (RPD) and Regenerative Medicine Advanced Therapy (RMAT) designations from FDA. In Europe, Lenmeldy is known as Libmeldy, where it has been approved by the European Commission (EC), UK Medicines and Healthcare products Regulatory Agency (MHRA), and Swiss Agency for Therapeutic Products (Swissmedic). (Source: Orchard Therapeutics Website, 18 and 20 March, 2024; and The Boston Globe, 21 March, 2024)