Cell and Gene Therapy

Gene Therapy for MLD Gets FDA Approval, Hefty Price Tag

Published March. 26. 2024
CGT Banner -Gene Therapy for MLD

Orchard Therapeutics, recently acquired by Kyowa Kirin, has set the wholesale acquisition cost (WAC) for their gene therapy at $4.25 million, the highest of any drug in the country. This price reflects the potential value the therapy may deliver to eligible patients and their families, as well as the long-term impact on overall healthcare utilization. The WAC was determined following an assessment by the Institute for Clinical and Economic Review, which set the health benefit price benchmark for the therapy at up to $3.94 million.

Metachromatic leukodystrophy (MLD), caused by a mutation on the ARSA gene, leads to a deficiency of the enzyme arylsulfatase A (ARSA), resulting in severe neurological damage and early death. Atidarsagene autotemcel, a one-time infusion, includes genetically modified hematopoietic stem cells with functional copies of the ARSA gene. The FDA approval was based on safety and efficacy data from clinical trials, showing promising outcomes in children with MLD.

Children treated with atidarsagene autotemcel showed improved survival rates and motor function compared to untreated children. Adverse reactions were reported, including febrile neutropenia, stomatitis, respiratory tract infections, and rash, among others. However, the therapy demonstrated significant potential in slowing the progression of MLD and improving patients’ quality of life.


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