National Centre for Pharmacoeconomics

National Centre for
Pharmacoeconomics

NCPE Ireland

OTL-200 Autologous CD34+ cells encoding arylsulfatase A (ARSA) gene (Libmeldy®). HTA ID: 21009

OTL-200 Autologous CD34+ cells encoding arylsulfatase A (ARSA) gene (Libmeldy®) is indicated 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.

 

NCPE Assessment Process Complete
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 the HSE 29/04/2021
Current status Awaiting HTA submission from Applicant