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|