ZEVRA THERAPEUTICS Business Summary

Zevra Therapeutics, Inc. (ZVRA) is a commercial-stage pharmaceutical company specializing in the development and commercialization of therapies for rare diseases with significant unmet medical needs. The company generates revenue through a diversified model comprising direct product sales, licensing agreements, and royalty streams. Its primary business segments are centered on rare disease therapeutics, led by its flagship product MIPLYFFA (arimoclomol) for Niemann-Pick disease type C and OLPRUVA for urea cycle disorders, alongside a royalty-bearing partnership with Corium for the ADHD treatment AZSTARYS. Zevra competes in the high-stakes orphan drug market against firms such as Sarepta Therapeutics, Aurinia Pharmaceuticals, and ANI Pharmaceuticals, positioning itself as an agile, commercial-ready player that leverages a proprietary prodrug platform and strategic acquisitions to capture niche patient populations. The leadership team is headed by President and CEO Neil F. McFarlane, a veteran biopharmaceutical executive with prior leadership roles at Adamas Pharmaceuticals and Retrophin (now Travere Therapeutics). Following a 2025 strategic reorganization, technical and scientific functions were consolidated under Chief Medical Officer Adrian Quartel, M.D., who previously served as CMO at Acer Therapeutics and Adamas. Other key executives include Chief Commercial Officer Joshua Schafer and Interim Principal Financial Officer Timothy Sangiovanni, who assumed his role in January 2026. The company was originally co-founded by Travis and Christal Mickle, the latter of whom departed in late 2024 as the firm transitioned toward late-stage commercial operations. Zevra is supported by a robust institutional investor base, including BlackRock and Vanguard, and is governed by a board chaired by Tamara A. Favorito, featuring industry experts like Dr. Alvin Shih and Wendy L. Dixon.