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New Gene Therapy for Rare MDL to Cost $4.25 Million


Lenmeldy is the first approved gene therapy to treat children with juvenile metachromatic leukodystrophy, a life-threatening inherited disease of the body’s metabolic system.

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.

The FDA had announced the approval of Lenmeldy on Monday. It 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.

Frank Thomas

Frank Thomas

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.

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.

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.

MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene, which results in the accumulation of sulfatides in the brain and other areas of the body. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat and see.

The safety and effectiveness of Lenmeldy was assessed based on data from 37 children who received Lenmeldy in two single-arm, open-label clinical trials and in an expanded access program. Children who received treatment with Lenmeldy were compared with untreated children.

In children with MLD, treatment with Lenmeldy significantly reduced the risk of severe motor impairment or death compared with untreated children. All children with pre-symptomatic late infantile MLD who were treated with Lenmeldy were alive at 6 years of age, compared with only 58% of children in the natural history group. At 5 years of age, 71% of treated children were able to walk without assistance. Eighty five percent of the children treated had normal language and performance IQ scores, which has not been reported in untreated children.

The most common side effects of Lenmeldy are fever and low white blood cell count, mouth sores, respiratory infections, rash, medical line infections, viral infections, fever, gastrointestinal infections and enlarged liver.

This story first appeared on Formulary Watch and has been edited.

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