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.
1180 Celebration Boulevard, Suite 103, Celebration, FL 34747, USA
Patents
Gamma-hydroxybutyrate delivering compounds and processes for making and using them
US-12251445-B2
View DetailsD-amphetamine compounds, compositions, and processes for making and using the same
US-12091386-B2
View DetailsCompositions comprising methylphenidate-prodrugs, processes of making and using the same
US-11505537-B2
View Details
Gamma-hydroxybutyrate delivering compounds and processes for making and using them
US-12251445-B2
View DetailsD-amphetamine compounds, compositions, and processes for making and using the same
US-12091386-B2
View DetailsCompositions comprising methylphenidate-prodrugs, processes of making and using the same
US-11505537-B2
View DetailsProducts
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
End-to-end clinical program management for rare-disease candidates including protocol design, site activation, and multi-phase trial execution.
Regulatory planning, submission preparation (IND/NDA/MAA), and formal agency engagement support including SPA management.
Commercial launch planning, market-access strategy, and partnership management for rare-disease products.
Design and running of expanded access programs and collection of real-world safety and effectiveness data to support regulatory and clinical claims.
Preparation of medical professional literature, conference presentations, and approved product literature.
Identification and execution of licensing, asset acquisitions, and commercial partnerships to expand pipeline and product reach.
End-to-end clinical program management for rare-disease candidates including protocol design, site activation, and multi-phase trial execution.
Regulatory planning, submission preparation (IND/NDA/MAA), and formal agency engagement support including SPA management.
Commercial launch planning, market-access strategy, and partnership management for rare-disease products.
Design and running of expanded access programs and collection of real-world safety and effectiveness data to support regulatory and clinical claims.
Preparation of medical professional literature, conference presentations, and approved product literature.
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
News & Updates
Neil F. McFarlane was appointed as President & CEO of Zevra Therapeutics.
Zevra's MIPLYFFA® (arimoclomol) received FDA approval as a treatment for Niemann-Pick Disease Type C.
Final results of the phase 2/3 NPC-002 48-month open-label extension trial.
Presenting a rescored 4-domain NPC Clinical Severity Scale.
MIPLYFFA® (arimoclomol) received FDA approval for the treatment of Niemann-Pick Disease Type C.
Neil F. McFarlane was appointed as President & CEO of Zevra Therapeutics.
Zevra's MIPLYFFA® (arimoclomol) received FDA approval as a treatment for Niemann-Pick Disease Type C.
Final results of the phase 2/3 NPC-002 48-month open-label extension trial.
Presenting a rescored 4-domain NPC Clinical Severity Scale.
MIPLYFFA® (arimoclomol) received FDA approval for the treatment of Niemann-Pick Disease Type C.