New Gene Therapy for Rare MDL to Cost $4.25 Million

Orchard Therapeutics has set the wholesale acquisition cost of its newly approved gene therapy, Lenmeldy (atidarsagene autotemcel), at $4.25 million for the one-time treatment. Company officials in a press release said this price reflects its clinical, economic and societal value. Lenmeldy is the first approved gene therapy to treat children with juvenile metachromatic leukodystrophy (MLD), a rare and life-threatening inherited disease of the body’s metabolic system.

“We are confident in the potential long-term clinical outcomes of Lenmeldy and will continue to work with public and private payers to structure outcomes-based and other types of innovative reimbursement models that appropriately balance the needs of patients and families for adequate access, healthcare systems for affordability, as well as support future research and development of treatments for ultra-rare diseases like MLD,” Frank Thomas, president and chief operating officer of Orchard Therapeutics, said in a press release.

Orchard Therapeutics has a patient support program for patients and their caregivers who will assist with both private and public insurance to access Lenmeldy. Infants with MLD qualify for government assistance so state Medicaid programs are expected to be part of the insurance mix.

Related: FDA Approves Gene Therapy for Rare Disease MDL

Five treatment centers with specialized expertise in transplant and the treatment of neurometabolic diseases are being activated to administer Lenmeldy.

Company officials said they relied on an analysis by the Institute for Clinical and Economic Review (ICER). But the actual price is above the range that ICER analysts had determined would meet the standards for cost-effectiveness. ICER’s analysis determined the health benefit price benchmark (HBPB) for Lenmeldy to be between $2.29 million and $3.94 million.

ICER’s assessment used a placeholder price of $2.8 million, and ICER noted that the gene therapy provides the most health benefit for presymptomatic disease. They stressed the need for newborn screening of MLD.

Sarah K. Emond

ICER President and CEO, Sarah K. Emond, said they encourage life sciences companies to rely on independent assessments of value to inform their pricing, they caution that the price chosen for Lenmeldy is $310,000 above the very top of ICER’s fair price range.

"Given payers are likely to cover treatment even at this high price, it is important to remember that every time prices exceed value, paying for them harms other unseen patients throughout our health system," she told Formulary Watch.

Orchard noted in its press release that it is currently conducting 10 prospective newborn screening studies for MLD with about 275,000 newborns screened to date. The data from these studies provide critical evidence to support applications for universal screening of MLD in the United States.

For infants showing signs of early disease, ICER officials said there is concern that the gene therapy “may carry the risk of hastening progression of physical and cognitive decline before stabilization occurs.” Because of this, ICER officials said they concluded there was a at least a small net health benefit.

This story has been updated to include comment from ICER.