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.”

References

  1. 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
  2. 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