CALLISTO was a phase I trial designed to assess the safety, tolerability and pharmacokinetics of omigapil for 12 weeks in 20 children with congenital muscular dystrophy (CMD) linked to COL6 or LAMA2.
The results of the study published this year showed good safety and a favourable pharmacokinetic profile of the product in these children. However, no evidence of efficacy was found, probably due, in the authors’ view, to the short duration of the trial.
As a reminder, Santhera Pharmaceuticals had announced that it would discontinue development of this compound in 2021.
Omigapil Community Statement
Cure CMD website, consulted on 07/09/2021
