Dive Brief:
- Orchard Therapeutics said Wednesday it will offer a new gene therapy to children with a rare, devastating disease at a record-setting wholesale price of $4.25 million.
- The therapy, Lenmeldy, won Food and Drug Administration approval on Monday to treat patients with early-onset metachromatic leukodystrophy, or MLD. The disease, which most often attacks infants between six months and two years of age, robs patients of the ability to walk, talk and function in the world, killing most of its earliest victims within five years of onset.
- Lenmeldy’s price tag will leapfrog those of the two most expensive gene therapies available in the U.S. Sarepta Therapeutics sells its Elevidys treatment for Duchenne muscular dystrophy for $3.2 million, while CSL and UniQure’s hemophilia treatment Hemgenix costs $3.5 million.
Dive Insight:
Gene therapy makers like Orchard are counting on insurers to recognize the value proposition of their products. Unlike traditional pharmaceuticals that might be taken regularly for the rest of a person’s life, gene therapies carry the promise of a long-lasting treatment that can alter the course of a disease and offer significant savings for the care needed to treat patients.
The argument doesn’t always work. Difficulty winning reimbursement forced gene therapy maker Bluebird bio to pull out of Europe in 2021. More recently, BioMarin has struggled with the launch of its hemophilia gene therapy, scaling back projections significantly. Orchard itself gave up on a gene therapy called Strimvelis in 2022.
But Orchard sees a different trajectory for Lenmeldy, in part because of its long-term data. Researchers have 12 years of follow-up on the earliest patients treated with Lenmeldy, the longest duration of any gene therapy launched in the U.S. Orchard also plans to develop outcomes- and value-based agreements designed to share risks with payers.
In studies, Orchard found that all patients with pre-symptomatic late infantile MLD treated with Lenmeldy were still alive at age six, compared with just 58% of such children historically. In addition, 71% of treated children could walk at age five without assistance and 85% had normal language and performance IQ scores. That has not been seen with untreated children.
“MLD places an enormous emotional and economic burden on families and caregivers – who face substantial wage loss and added expenses each year as the disease progresses – all while dealing with the unquantifiable anguish of losing their child,” Bennett Smith, Orchard’s senior vice president and general manager of North America, said in the company’s statement. Gene therapy can be “transformative,” he said.
In Wednesday’s release, Orchard highlighted the findings of the Institute for Clinical and Economic Review, which found that Lenmeldy had the highest value-based price of any treatment it has evaluated. Still, Orchard priced its therapy above the $2.3 million to $3.9 million cost-effectiveness threshold determined by that group.
Orchard, which was acquired by Kyowa Kirin this year, is setting up five treatment centers across the U.S. to administer Lenmeldy. The first, in Minnesota, is in the final stages of qualification and has already treated several children on a compassionate use basis. Four others, in Atlanta, Philadelphia, San Francisco and Texas, are in the process of qualification.
