Our expertise, elegant solutions, and transparent data narratives have produced a late-stage rare disease clinical pipeline and two partnered commercial products.
The below product candidates are under investigation.
EMA MAA Submission: Targeting H2 2025
New chemical entity for the treatment of COL3A1-positive VEDS.
Phase 3 trial ongoing
Granted orphan drug designation and breakthrough therapy designation by the U.S. FDA. Celiprolol is a New Chemical Entity currently in Phase 3 development for the treatment of COL3A1-positive VEDS patients to potentially reduce the risk of arterial and other hollow organ clinical events. The Phase 3 protocol is being conducted under a Special Protocol Assessment (SPA) agreement with the U.S. FDA. For more information.
Prodrug candidate for IH and narcolepsy type I & II
Phase 3 trial ready
Phase 3 trial potential
Serdexmethylphenidate (SDX) is Zevra’s proprietary prodrug of d-methylphenidate (d-MPH) and the sole active pharmaceutical ingredient (API) in KP1077, Zevra’s investigational product candidate for the treatment of idiopathic hypersomnia (IH) and narcolepsy, respectively. IH is a rare neurological sleep disorder that can exhibit symptoms similar to narcolepsy. Narcolepsy is a chronic neurological disorder that affects the brain's ability to control sleep-wake cycles.
KP1077 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of IH and may be eligible for expedited approval pathways. The U.S. Drug Enforcement Agency (DEA) has classified SDX as a Schedule IV controlled substance based on evidence suggesting SDX has a lower potential for abuse when compared to d-MPH, a Schedule II controlled substance.
Zevra completed a multicenter, dose-optimizing, double-blind, placebo-controlled, randomized-withdrawal Phase 2 clinical trial to evaluate the safety and efficacy of KP1077 as a treatment for IH.
Phase 1 clinical trial under the narcolepsy IND was completed for KP1077 in healthy volunteers to support clinical development of both narcolepsy and IH programs. This study provided additional information for optimizing the dosing regimen of KP1077 to assess the pharmacokinetics of SDX when dosed in the morning versus at night.
Learn MoreThese product candidates are under investigation and their safety and efficacy have not been established. There is no guarantee that these products will receive health authority approval or become commercially available for the uses being investigated.
NOW ENROLLING COL3A1 positive VEDS patients (15 years and older)
Study ACER002-301 is being conducted by Zevra to evaluate the efficacy and safety of celiprolol.
Celiprolol is an adrenoceptor modulator believed to decrease mechanical stress on the vascular wall of large arteries and hollow organs such as the uterus. It is an investigational medication for the treatment of Vascular Ehlers Danlos Syndrome (VEDS).
The clinical study intends to enroll 150 COL3A1 positive VEDS patients. 50 patients will receive placebo and 100 patients will receive celiprolol up to 400 mg/day (200mg bid). The study will conclude when 46 qualifying events (fatal or non-fatal cardiac event such as rupture of dissection, uterine rupture, colon rupture and/or unexplained sudden death) have been reported.