Zevra Therapeutics, Inc.
Zevra Therapeutics is a nimble rare disease therapeutics company dedicated to developing and delivering therapies that address significant unmet needs in rare diseases. With a focus on science and innovation, they involve key stakeholders including physicians, patients, and advocacy groups to design better clinical programs. Their mission is to give every promising rare disease therapy a fighting chance to reach patients and improve their quality of life.
Industries
Nr. of Employees
small (1-50)
Zevra Therapeutics, Inc.
Orlando, Florida, United States, North America
Products
MIPLYFFA (arimoclomol) — commercial product
Oral capsule indicated, in combination with miglustat, for neurological manifestations of Niemann‑Pick disease type C in patients aged 2 years and older.
OLPRUVA (sodium phenylbutyrate) — commercial product
Dual-coated oral suspension indicated in the U.S. for treatment of certain urea cycle disorders involving CPS, OTC, or AS deficiencies.
AZSTARYS (serdexmethylphenidate + dexmethylphenidate) — commercial product (royalty partner)
Prodrug-based stimulant product approved for the treatment of ADHD in patients six years and older; company collects royalties and milestones through a commercial partnership.
Arimoclomol (pipeline / regulatory submissions)
Orally delivered small-molecule candidate developed for Niemann‑Pick disease type C; subject of Phase 3 development and regulatory filings.
Celiprolol (investigational New Chemical Entity for VEDS)
New chemical entity under Phase 3 evaluation for COL3A1-positive vascular Ehlers‑Danlos syndrome to potentially reduce risk of arterial and other hollow‑organ events.
KP1077 (serdexmethylphenidate prodrug candidate for sleep disorders)
Prodrug investigational product candidate containing a prodrug of d‑methylphenidate being studied for idiopathic hypersomnia and narcolepsy; clinical program includes Phase 1 and Phase 2 studies with Phase 3 readiness.
MIPLYFFA (arimoclomol) — commercial product
Oral capsule indicated, in combination with miglustat, for neurological manifestations of Niemann‑Pick disease type C in patients aged 2 years and older.
OLPRUVA (sodium phenylbutyrate) — commercial product
Dual-coated oral suspension indicated in the U.S. for treatment of certain urea cycle disorders involving CPS, OTC, or AS deficiencies.
AZSTARYS (serdexmethylphenidate + dexmethylphenidate) — commercial product (royalty partner)
Prodrug-based stimulant product approved for the treatment of ADHD in patients six years and older; company collects royalties and milestones through a commercial partnership.
Arimoclomol (pipeline / regulatory submissions)
Orally delivered small-molecule candidate developed for Niemann‑Pick disease type C; subject of Phase 3 development and regulatory filings.
Celiprolol (investigational New Chemical Entity for VEDS)
New chemical entity under Phase 3 evaluation for COL3A1-positive vascular Ehlers‑Danlos syndrome to potentially reduce risk of arterial and other hollow‑organ events.
KP1077 (serdexmethylphenidate prodrug candidate for sleep disorders)
Prodrug investigational product candidate containing a prodrug of d‑methylphenidate being studied for idiopathic hypersomnia and narcolepsy; clinical program includes Phase 1 and Phase 2 studies with Phase 3 readiness.
Services
Clinical development and trial execution
End-to-end clinical program management for rare-disease candidates including protocol design, site activation, and multi-phase trial execution.
Regulatory strategy and submission support
Regulatory planning, submission preparation (IND/NDA/MAA), and formal agency engagement support including SPA management.
Commercialization and market-access services
Commercial launch planning, market-access strategy, and partnership management for rare-disease products.
Real-world evidence and expanded access program operation
Design and running of expanded access programs and collection of real-world safety and effectiveness data to support regulatory and clinical claims.
Medical affairs and scientific communications
Preparation of medical professional literature, conference presentations, and approved product literature.
Partnerships and business development
Identification and execution of licensing, asset acquisitions, and commercial partnerships to expand pipeline and product reach.
Clinical development and trial execution
End-to-end clinical program management for rare-disease candidates including protocol design, site activation, and multi-phase trial execution.
Regulatory strategy and submission support
Regulatory planning, submission preparation (IND/NDA/MAA), and formal agency engagement support including SPA management.
Commercialization and market-access services
Commercial launch planning, market-access strategy, and partnership management for rare-disease products.
Real-world evidence and expanded access program operation
Design and running of expanded access programs and collection of real-world safety and effectiveness data to support regulatory and clinical claims.
Medical affairs and scientific communications
Preparation of medical professional literature, conference presentations, and approved product literature.
Partnerships and business development
Identification and execution of licensing, asset acquisitions, and commercial partnerships to expand pipeline and product reach.
Expertise Areas
- Clinical trial management (Phase 1–3)
- Regulatory strategy and submissions (IND/NDA/MAA)
- Orphan drug and expedited pathway navigation
- Prodrug chemistry and development
Key Technologies
- Prodrug design and development
- Pharmacokinetic modeling and bioavailability analysis
- Randomized-withdrawal clinical trial design
- Open-label extension study methods