AR-42 is a broad-spectrum deacetylation inhibitor of both histone and non-histone proteins.
The orally available inhibitor has demonstrated greater potency and activity in solid tumours and hematological malignancies when compared in preclinical studies to vorinostat.
Arno chief executive officer and president Glenn Mattes said, "The EMA’s orphan-drug designation represents another important step in our development program for AR-42 that will help us pursue its clinical development for the treatment in this rare and debilitating disease."
AR-42 is currently being studied in a Phase I/IIa trial with relapsed or refractory hematologic malignancies patients and solid tumour patients.
NF2 is a genetic disorder characterized by the growth of noncancerous tumours in the brain and spinal cord, juvenile cataracts and neurofibromas of the skin.