Orchard Therapeutics outlines U.S. launch plans for Lenmeldy (atidarsagene autotemcel), the only approved therapy for children with early-onset metachromatic leukodystrophy.

The WAC was determined following the completion of a comprehensive Health Technology Assessment (HTA) by the independent, non-profit organization, the Institute for Clinical and Economic Review (ICER) which determined the health benefit price benchmark (HBPB) for Lenmeldy to be up to $3.94 million at the $150,000 per Equal Value Life Year (evLY) threshold from a modified societal perspective.

Lenmeldy is being made available to eligible patients through a network of Qualified Treatment Centers (QTCs) in key regions throughout the United States to minimize the travel burden on patients and their families. Five treatment centers with specialized expertise in transplant and the treatment of neurometabolic diseases, like MLD, are being activated. One of those centers, the M Health Fairview Masonic Children’s Hospital in Minnesota, is in the final stages of qualification. Several eligible children with MLD from the U.S. have already been treated at the center on a compassionate use basis with drug product supplied by Orchard Therapeutics manufactured using commercial processes.

Four additional regional centers geographically dispersed throughout the United States, including Children’s Healthcare of Atlanta, Children's Hospital of Philadelphia, Texas Children’s Hospital, and UCSF Benioff Children’s Hospital San Francisco, are in the process of becoming fully qualified.

As with many rare, life-threatening pediatric diseases, early detection and diagnosis is key to ensuring the best possible outcomes for patients, and Orchard Therapeutics supports efforts to expand newborn screening (NBS) for diseases like MLD which meet Wilson and Jungner criteria. Currently, ten prospective NBS studies for MLD are active throughout the U.S., Europe and the Middle East, with approximately 275,000 newborns screened to date. The data from these studies provide critical evidence to support applications for universal screening of MLD in the U.S. and around the world.

Utilizing results from such studies, a multi-stakeholder working group is finalizing a nomination to add MLD to the U.S. Recommended Uniform Screening Panel (RUSP), a national guideline for NBS comprising a list of medical conditions for which the federal government recommends all newborns receive screening. States use the RUSP to help them decide which conditions to include in their NBS panels. Based on current timelines and assumptions, Orchard Therapeutics expects the nomination will be submitted in mid-year 2024.