Atidarsagene (Libmeldy®). HTA ID: 21009

Assessment Status NCPE Assessment Process Complete
HTA ID 21009
Drug Atidarsagene autotemcel
Brand Libmeldy®
Indication For the treatment of metachromatic leukodystrophy characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity, in children with late infantile or early juvenile forms without clinical manifestations of the disease, and in children with the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline.
Assessment Process
Rapid review commissioned 22/03/2021
Rapid review completed 12/04/2021
Rapid review outcome A full HTA is recommended to assess the clinical effectiveness and cost effectiveness of OTL-200 compared with the current standard of care.
Full pharmacoeconomic assessment commissioned by HSE 29/04/2021
Full submission received from Applicant 17/12/2021
NCPE assessment completed 30/09/2022
NCPE assessment outcome Following the NCPE/Benaluxa assessment the NCPE recommends that atidarsagene autotemcel not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatment*.

NCPE/Benaluxa Joint Technical Summary

Plain English Summary

Ireland, Belgium and the Netherlands, as members of the Beneluxa Initiative, have successfully concluded joint pricing negotiations for atidarsagene (Libmeldy®). The HSE has approved reimbursement following joint confidential price negotiations, January 2024.

Further information is available here.

*This recommendation should be considered while also having regard to the criteria specified in the Health (Pricing and Supply of Medical Goods) Act 2013.