A report from analysts at Jefferies suggested that new screenings for metachromatic leukodystrophy and Duchenne muscular dystrophy could bump sales of the gene therapy Libmeldy by more than $100 million.
Health Secretary Robert F. Kennedy Jr.’s recent move to add two new conditions to standard newborn screening protocols could put money in Kyowa Kirin’s coffers, according to a report from Jefferies analysts published Tuesday.
Earlier this week, Kennedy added metachromatic leukodystrophy (MLD) and Duchenne muscular dystrophy to the list of diseases included on the federal government’s Recommended Uniform Screening Panel (RUSP), a list of diseases that the Department of Health and Human Services (HHS) suggests that newborns be tested for.
While states are not bound by the list, Jefferies anticipates that Kyowa, which sells a one-time gene therapy for MLD called Libmeldy through its subsidiary Orchard Therapeutics, could benefit. Adding MLD to the RUSP “is likely to accelerate the adoption of MLD screening and boost demand for the gene therapy treatment, Libmeldy,” the analysts wrote, noting that the treatment carries a price tag of $4.25 million.
Finding More Patients
The FDA approved Libmeldy in 2024 and Orchard began selling it in the U.S. this year. Jefferies estimated that four patients in the country will be treated this year, amounting to ¥2.1 billion ($13.3 million). With the change to RUSP, Jefferies suggested that at least 14 states, accounting for more than half of the country’s population, will screen for MLD, possibly bumping the total treated in the U.S. to 40 each year. That could add ¥18 billion ($114.3 million) in sales for Libmeldy.
Jefferies estimated that worldwide, Libmeldy’s sales will hit ¥6.9 billion ($43.8 million) this year.
Patients with MLD, a rare lysosomal storage disease that effects the nervous system, most often die between ages five and eight, and though Libmeldy doesn’t completely block symptoms, it does extend survival and slow progression when administered early, according to the Jefferies report.
Orchard commended the recommended inclusion of MLD on the list in a statement released Tuesday. CEO Bobby Gaspar cautioned, however, that while “the addition of MLD to the RUSP is a monumental step toward enabling newborn screening in the U.S., implementation now must happen at the state-level, a historically multi-year process inhibited by the lack of adequate federal and state funding.”
Debra Miller, the founder and CEO of research and advocacy group CureDuchenne, was pleased with Kennedy’s announcement as well. “We applaud HHS for this decision, which marks a breakthrough moment for our community,” she said in a statement to BioSpace. “Early diagnosis offers families a critical window to access care and emerging treatments when they may be most effective.”
Libmeldy is the only FDA-approved treatment for children with MLD on the market. It provides patients with a functional copy of the ARSA gene, which is mutated in the condition.
“This approval represents important progress in the advancement and availability of effective treatments, including gene therapies, for rare diseases,” then-director of the Office of Therapeutic Products Nicole Verdun said at the time of the therapy’s approval.
