Hyperion Receives Special Protocol Assessment Approval From FDA

Hyperion has reached an agreement with FDA (under the Special Protocol Assessment process) for the pivotal phase-III trial of investigational product HPN-100 (glycerol phenylbutyrate) in adults with urea cycle disorders (UCD).

The four-week, multi-center, randomized, double-blind, cross-over study in adults with urea cycle disorders, is designed to evaluate the non-inferiority of HPN-100 to BUPHENYL (sodium phenylbutyrate). The primary efficacy measure is blood ammonia, assessed as 24-hour area under the curve on days 14 and 28. The study intends to enroll approximately 44 adults, and all subjects completing the study to be eligible to enter a 12-month, open label safety study.

Bruce Scharschmidt, Chief Medical Officer of Hyperion, said: We appreciate the input from FDA on the pivotal Phase III study protocol and look forward to continuing development of HPN-100 for urea cycle disorder patients, a population with limited treatment options.