The company has recently announced results from its Phase II study and intends to initiate a Phase III clinical program in this indication in the second half of 2009.
HPN-100 is a pre-pro-drug of phenylacetic acid which is the active moiety of Buphenyl, reportedly the only therapy currently FDA-approved as adjunctive therapy for the chronic management of patients with the most prevalent urea cycle deficiencies including those related to carbamylphosphate synthetase, ornithine transcarbamylase and argininosuccinic acid synthetase. HPN-100 is dosed orally in liquid form.
Donald Santel, CEO of Hyperion, said: “Receiving orphan drug designation for HPN-100 in the treatment of urea cycle disorders is an important milestone and provides meaningful benefits to the company as we work to complete clinical trials for this investigational compound.”