Aerial BioPharma, a biopharmaceutical company, has won FDA orphan drug designation for its narcolepsy drug in development.
The issuance of designation from the FDA follows the completion of a Phase IIa study in which N05 demonstrated statistically significant results on the primary and secondary efficacy endpoints in patients with narcolepsy.
The company expects to begin narcolepsy treatment in a Phase IIb trial in September 2012 and anticipates results in mid-2013.
Aerial BioPharma chief executive officer Moise Khayrallah said the positive efficacy and safety data from the Phase IIa trial show promise for controlling the symptoms of narcolepsy.
"Achieving the orphan drug status is a key milestone and another testament to the team's expertise in identifying and rapidly developing drug candidates with a high probability of success and a clear regulatory path to approval," Khayrallah added.
Aerial BioPharma, based in Morrisville, North Carolina, is a privately-held biopharmaceutical company focused on developing biologics and small molecules for conditions in the central nervous system.