We are dedicated to developing transformational, patient-focused therapies for rare diseases with limited or no treatment options available, or treatment areas with significant unmet needs. Our expertise and science-driven approach has produced a late-stage rare disease clinical pipeline and multiple commercial products.
The below product candidates are under investigation.
Filed Marketing Authorization Application with the European Medicines Agency.
Arimoclomol is under review by the European Medicines Agency (EMA) for the treatment of Niemann-Pick disease type C (NPC). Arimoclomol increases the activation of the transcription factors EB (TFEB) and E3 (TFE3) resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. Arimoclomol has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. The clinical significance of these findings is not fully understood. In the pivotal Phase 3 trial, arimoclomol halted disease progression compared to placebo in patients taking miglustat over the twelve month duration of the trial when measured by the only validated disease progression measurement tool, the NPC Clinical Severity Scale. Arimoclomol has also received Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC.
New chemical entity for the treatment of COL3A1-positive VEDS.
Celiprolol is a selective adrenoceptor modulator, or a unique type of beta blocker, believed to decrease mechanical stress on the vascular wall of large arteries and hollow organs. It is 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 DiSCOVER trial is being conducted under a Special Protocol Assessment (SPA) agreement with the U.S. FDA. Celiprolol has been granted orphan drug designation and breakthrough therapy designation by the U.S. FDA. Click here to learn more about the DiSCOVER trial. Click here for the Celiprolol Fact Sheet.
Prodrug candidate for IH and narcolepsy type I & II
Phase 3 trial ready
Phase 3 trial potential
KP1077 is an 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.
A 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 trial provided additional information for optimizing the dosing regimen of KP1077.
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)
The DiSCOVER trial is being conducted by Zevra to evaluate the efficacy and safety of celiprolol.
Celiprolol is a selective 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 trial 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 trial 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.