Press release
KemPharm Receives FDA Orphan Drug Designation for Serdexmethylphenidate (SDX) for the Treatment of Idiopathic Hypersomnia (IH)
Phase 2 clinical trial investigating KP1077 in patients with IH expected to initiate prior to year-end 2022 CELEBRATION, Fla., Nov. 18, 2022 (GLOBE NEWSWIRE)
About this update from Zevra Therapeutics, Inc.
[{"type":"text","content":"Phase 2 clinical trial investigating KP1077 in patients with IH expected to initiate prior to year-end 2022 CELEBRATION, Fla., Nov. 18, 2022 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NasdaqGS: KMPH) (KemPharm, or the Company), a biotechnology company focused on the discovery, development and commercialization of novel treatments for rare central nervous system (CNS) and neurodegenerative diseases, lysosomal storage disorders and related treatment areas, today announced that the U.S. Food and Drug Administration (FDA) has granted the Orphan Drug Designation to serdexmethylphenidate (SDX), KemPharm’s proprietary prodrug of d-methylphenidate (d-MPH), for the treatment of idiopathic hypersomnia (IH), a rare neurological sleep disorder. SDX is the sole active pharmaceutical ingredient (API) in KP1077, KemPharm’s lead clinical candidate being developed as a treatment for IH and narcolepsy. KemPharm expects to initiate a Phase 2 clinical trial of KP1077 in patients with IH prior to year-end 2022 and a second trial in patients with narcolepsy in 2023. “We appreciate the FDA’s decision to grant Orphan Drug Designation to SDX for IH, a rare sleep disorder characterized by multiple, debilitating symptoms for which few treatment options exist,” stated Travis Mickle, Ph.D., President and Chief Executive Officer of KemPharm. “This regulatory milestone comes at an important juncture in the ongoing development of KP1077 as we prepare to initiate the Phase 2 clinical trial in IH before year-end 2022. We believe KP1077 could provide an improved treatment option for patients with IH and other sleep disorders by addressing the most debilitating symptoms of IH, 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.” FDA Orphan Drug Designation may be granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States, or a patient population greater than 200,000 individuals in the United States and when there is no reasonable expectation that the cost of developing and making available the drug in the United States will be recovered from sales in the United States for that drug. If a product that has Orphan Drug Desig...