Zevra Therapeutics, Inc. ZVRA
Zevra Therapeutics, Inc. (Nasdaq: ZVRA) is a rare genetic drug developer. Its lead program, MIPLYFFA (arimoclomol), is in Phase 3 for Niemann-Pick disease type C (NPC). Its next catalyst on record is Celiprolol: FDA Type C follow-up meeting to discuss regulatory options to potentially accelerate development program (Q4 2026).
Upcoming catalysts
No catalyst is dated to the next 30 days or to an FDA action date.
Next further out: Celiprolol — FDA Type C follow-up meeting to discuss regulatory options to potentially accelerate development program (Q4 2026). 2 catalysts on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| MIPLYFFA (arimoclomol) | Niemann-Pick disease type C (NPC) | small molecule | Approved |
| OLPRUVA (sodium phenylbutyrate) | Urea Cycle Disorders (UCD) | small molecule | Approved |
| Celiprolol | Vascular Ehlers-Danlos Syndrome (VEDS) | small molecule | Phase 3 |
Recent results and decisions
- Nov 3, 2026n/a Zevra enrolled six patients in Q3 2026 in the Phase 3 DiSCOVER trial of celiprolol, bringing total enrollment to 72 patients with four confirmed events.
Enrolled six patients in the event-driven Phase 3 DiSCOVER trial of celiprolol for the treatment of Vascular Ehlers-Danlos Syndrome during Q3 2026, bringing the total number of enrolled patients to 72, with a total of four confirmed events.
Filing - Nov 3, 2026n/a Zevra met with FDA to discuss potential supplemental NDA for MIPLYFFA in infants less than 24 months of age following publication of pediatric data.
Following the publication of its pediatric data in Molecular Genetics and Metabolism Reports, Zevra met with the U.S. Food and Drug Administration (FDA) to discuss a potential supplemental New Drug Application (sNDA) for MIPLYFFA in infants less than 24 months of age.
Filing
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
- Expected move for each dated catalyst, from comparable past events
- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 3 rare genetic companies
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.