The Food and Drug Administration (FDA) has granted Orphan Drug designation to serdexmethylphenidate (KP1077) for the treatment of idiopathic hypersomnia.
Idiopathic hypersomnia is a rare neurological sleep disorder with symptoms that include excessive daytime sleepiness, extreme difficulty waking, severe brain fog, and narcolepsy. Serdexmethylphenidate is a proprietary prodrug of d-methylphenidate.
The cardiovascular safety of serdexmethylphenidate was evaluated in a phase 1 trial that included 15 volunteers. The higher dose formulation of serdexmethylphenidate was found to be safe and well tolerated when compared with racemic methylphenidate. Based on these findings, a phase 2 clinical trial evaluating serdexmethylphenidate in patients with idiopathic hypersomnia is expected to begin before the end of 2022. A second clinical trial enrolling patients with narcolepsy is planned for 2023.
“We appreciate the FDA’s decision to grant Orphan Drug designation to [serdexmethylphenidate] for [idiopathic hypersomnia], a rare sleep disorder characterized by multiple, debilitating symptoms for which few treatment options exist,” said Travis Mickle, PhD, President and CEO of KemPharm. “We believe [serdexmethylphenidate] could provide an improved treatment option for patients with [idiopathic hypersomnia] and other sleep disorders by addressing the most debilitating symptoms of [idiopathic hypersomnia], including excessive daytime sleepiness, extreme difficulty waking up (sleep inertia), severe brain fog, and falling asleep unintentionally and/or at inappropriate times, even after adequate or prolonged nighttime sleep.”
- KemPharm receives FDA Orphan Drug designation for serdexmethylphenidate (SDX) for the treatment of idiopathic hypersomnia (IH). News release. KemPharm, Inc. Accessed November 18, 2022. https://www.globenewswire.com/news-release/2022/11/18/2559035/16626/en/KemPharm-Receives-FDA-Orphan-Drug-Designation-for-Serdexmethylphenidate-SDX-for-the-Treatment-of-Idiopathic-Hypersomnia-IH.html
- KemPharm announces positive topline data from phase 1 clinical trial evaluating cardiovascular safety of serdexmethylphenidate (SDX). News release. KemPharm, Inc. September 28, 2022. Accessed November 18, 2022. https://investors.kempharm.com/news-releases/news-release-details/kempharm-announces-positive-topline-data-phase-1-clinical-trial