Positive Opinion for Orphan Drug Designation

2009-12-03

Medesis Pharma's treatment for Huntington's disease has received a positive opinion for orphan drug designation from the Committee for Ophan Medicinal Products (COMP).    Lithium citrate tetrahydrate (in reverse-micelle formulation) for treatment of Huntington's disease (NP03)  fulfilled the requirements for orphan drug designation based upon the level of prevalence of the disease in the European Community, and on the medical plausibility data based upon studies in a relevant transgenic mouse model.  The neuroprotective activity shown in the mouse model at low doses of lithium, highlighted the unique delivery properties of the reverse micelle formulation, Aonys®, for lithium and other metals