Orchard Therapeutics, recently acquired by Kyowa Kirin to accelerate the delivery of new gene therapies globally, announced on March 20, 2024, its U.S. commercial launch plans for Lenmeldy™ (atidarsagene autotemcel), the first FDA-approved therapy for children with early-onset metachromatic leukodystrophy (MLD), a rare and fatal neurometabolic disorder. MLD affects approximately 1 in 100,000 live births in the U.S., leading to severe neurological damage and a drastically shortened life expectancy. Lenmeldy™ aims to treat MLD at its genetic root by introducing functional copies of the ARSA gene into a patient’s hematopoietic stem cells, potentially halting or slowing the disease’s progression with a single treatment.
To make this therapy accessible and reduce the travel burden on patients and their families, Orchard Therapeutics is qualifying five specialized treatment centers across the U.S. and has introduced the Orchard Assist patient services program to offer individualized support throughout the treatment journey. The company has set the wholesale acquisition cost of Lenmeldy™ at $4.25 million, reflecting its significant clinical, economic, and societal benefits, including the potential to reduce healthcare utilization and improve patients’ life opportunities.
Orchard Therapeutics is committed to ensuring broad, expedient, and sustainable access to Lenmeldy™ through innovative outcomes- and value-based agreements with both private and government insurers. This approach aims to balance patient needs with healthcare system affordability and support future research and development. FDA approval of Lenmeldy™ was based on data from clinical studies showing significant improvements in survival and functional outcomes compared to natural history data, with a safety profile supported by long-term follow-up data.
How Lenmeldy™ and New Treatment Centers Transform the Fight Against MLD?
To provide specialized care for patients with neurometabolic diseases like MLD, Qualified Treatment Centers (QTCs) are being established in key U.S. regions, including M Health Fairview Masonic Children’s Hospital in Minnesota among others. Additionally, the Orchard Assist program is designed to help patients and families navigate the treatment process, offering assistance from experienced case managers.
Orchard Therapeutics also supports efforts to expand newborn screening for diseases like MLD, with ongoing studies already screening approximately 275,000 newborns. This early detection is crucial for achieving the best possible outcomes for patients. The launch of Lenmeldy™ represents a significant advancement in the treatment of MLD, offering new hope to affected families and setting a new standard for the value of gene therapies in treating rare diseases.
Resource: Orchard Therapeutics, March 20, 2024
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