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 neurological manifestations of Niemann-Pick disease type C (NPC), in combination with miglustat. Its next catalyst on record is celiprolol: Follow-up FDA meeting on regulatory options to potentially accelerate development program (after Type C meeting in 2026-Q1) (H2 2026).
Upcoming catalysts
No catalyst is dated to the next 30 days or to an FDA action date.
Next further out: celiprolol — Follow-up FDA meeting on regulatory options to potentially accelerate development program (after Type C meeting in 2026-Q1) (H2 2026). 1 catalyst 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 (FDA approved Sept 20, 2024; EU MAA received negative CHMP opinion July 2026, re-examination requested) | small molecule | Approved |
| OLPRUVA (sodium phenylbutyrate oral suspension) | urea cycle disorders (FDA approved Dec 2022; commercial efforts scaled back) | small molecule | Approved |
| celiprolol (DiSCOVER) | vascular Ehlers-Danlos syndrome (COL3A1 mutation); Phase 3, 66 of 150 patients enrolled; Breakthrough Therapy | small molecule | Phase 3 |
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.