The Danish biotechnology company, Orphazyme, announced in early May of this year that enrollment was completed for its lead study program for Niemann-Pick type C disease.
Niemann-Pick disease is a term used to describe a group of rare lysosomal storage disorders that are characterized by the buildup of fatty substances in the organs.
Currently, treatment of the condition is focused on symptom management, so there is a need in the rare disease community for a better therapy option. To learn more about Niemann-Pick disease, click here.
Orphazyme is addressing this unmet need its new pipeline treatment, arimoclomol, in a double-blind placebo interventional study. The goal of this trial is to assess the safety and efficacy of arimoclomol when it is administered alongside the patient’s current best standard of care that has been provided by his/her medical professional team.
Arimoclomol works by activating molecular chaperones, thus stimulating normal cellular protein repair pathways. Damaged proteins are thought to play a role in the onset of Niemann-Pick disease, so arimoclomol is an exciting option for treating this disease at its core.
The trial, which is aimed to terminate in the second quarter of 2018, has the ultimate goal of diminishing the overall disease severity of Niemann-Pick without any major side-effects or intolerability.
Hopefully in a year from now, arimoclomol will be proven effective and safe, and the Niemann-Pick disease community will have a new option to lighten the burden of their rare condition.
To learn more about arimoclomol in the meantime, click here!