Zevra Therapeutics, Inc. is a commercial-stage rare disease company that combines science, data and patient needs to create therapies for diseases with limited or no treatment options. The firm focuses on the treatment of rare neurological and sleep conditions such as certain Urea Cycle Disorders, Niemann Pick Disease Type C and Idiopathic Hypersomnia. The company receives royalties for AZSTARYS from its commercial partner Corium. The company was founded by Christal M. M. Mickle and Travis C. Mickle on October 30, 2006 and is headquartered in Celebration, FL.
Clinical Trials
3
0 active
Approvals
0
Total approvals
Agencies
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Founded
2006
Completed
3
100.0%
No approval data available
- Zevra Therapeutics has submitted U.S. Patent No. 11,707,456 for MIPLYFFA (arimoclomol) to the FDA for Orange Book listing, extending patent protection to November 19, 2041. - The Orange Book listing strengthens market exclusivity by enabling an automatic stay on generic approvals if a generic filer challenges the patent. - MIPLYFFA is approved for Niemann-Pick disease type C (NPC), an ultra-rare, progressive neurodegenerative disease with significant unmet medical need. - A separate patent term extension application for U.S. Patent No. 11,045,460 remains pending with the U.S. Patent and Trademark Office.
- More than three pharmaceutical companies are actively developing over four promising hypersomnia treatment candidates, signaling growing innovation in sleep disorder management. - Avadel Pharmaceuticals received FDA orphan drug designation in June 2025 for Lumryz, an extended-release sodium oxybate therapy for idiopathic hypersomnia. - Alkermes initiated a Phase II trial in April 2025 evaluating ALKS 2680, a selective orexin receptor modulator, in patients with idiopathic hypersomnia across global sites. - Axsome Therapeutics reported positive Phase III results in November 2024 for AXS-12 (reboxetine), demonstrating significant reductions in cataplexy episodes.
- Zevra Therapeutics has executed an exclusive expanded access distribution agreement with Ireland-based Uniphar to broaden access to MIPLYFFA (arimoclomol) for Niemann-Pick Disease Type C patients in select territories outside Europe. - MIPLYFFA, used in combination with miglustat, is the only treatment shown to halt disease progression in NPC, with improvement seen at week 12 and durable effects lasting more than five years. - The drug is currently approved by the FDA and commercially available in the U.S., while a Marketing Authorization Application is under review by the European Medicines Agency. - Clinical data from more than 270 NPC patients worldwide demonstrates the most expansive clinical development program in NPC to date, with over five years of patient experience across multiple studies.
- XOMA Royalty Corporation reported robust financial performance in Q3 2025, receiving $43.9 million in royalties and milestones from partners in the first nine months of 2025, including $14.3 million in Q3 royalties. - The company completed strategic acquisitions of Turnstone Biologics and HilleVax while announcing planned acquisitions of LAVA Therapeutics and Mural Oncology to expand its royalty portfolio. - Key pipeline developments include Zevra Therapeutics submitting a Marketing Authorization Application to the EMA for arimoclomol in Niemann-Pick Type C disease and Rezolute Bio expecting Phase 3 ersodetug trial results in December 2025. - XOMA Royalty achieved net income of $14.1 million in Q3 2025 compared to a net loss of $17.2 million in the same period of 2024, demonstrating improved operational performance.
- Zevra Therapeutics achieved Q2 2025 net revenue of $25.9 million, driven primarily by $21.8 million in product revenue, marking significant growth from $4.4 million in Q2 2024. - The company completed the sale of its Pediatric Rare Disease Priority Review Voucher for $150 million, providing non-dilutive capital to support commercial launches and development programs. - Zevra submitted a Marketing Authorisation Application to the European Medicines Agency for arimoclomol (MIPLYFFA) to treat Niemann-Pick Disease Type C, seeking to expand access beyond the U.S. market. - The company enrolled 7 additional patients in its Phase 3 DiSCOVER trial of celiprolol for Vascular Ehlers-Danlos Syndrome, bringing total enrollment to 39 patients.
- Zevra Therapeutics published long-term data showing MIPLYFFA (arimoclomol) sustained disease progression reduction for at least 5 years in Niemann-Pick disease type C patients. - The 48-month open-label extension study included more than 270 patients worldwide, with some receiving treatment for up to seven years. - Results demonstrated no new safety concerns and aligned with the pivotal Phase 2/3 trial that showed MIPLYFFA halted disease progression compared to placebo. - MIPLYFFA is approved by the FDA for use in combination with miglustat for treating neurological manifestations of NPC in patients 2 years and older.
- Bavarian Nordic announced the sale of its Priority Review Voucher for $160 million, awarded following FDA approval of its chikungunya vaccine VIMKUNYA in February 2025. - The transaction is expected to close in Q3 2025 and will positively impact the company's EBITDA, with the National Institutes of Health receiving 20% of proceeds under a licensing agreement. - The sale reflects an uptick in PRV values in 2025, with recent transactions ranging from $150-160 million compared to the historical average of around $100 million. - Priority Review Vouchers reduce FDA drug review times from ten months to six months and are awarded for treatments addressing neglected tropical diseases, rare pediatric conditions, or material threat medical countermeasures.
- The median annual list price for newly launched pharmaceuticals in the U.S. has more than doubled to $370,000 in 2024 compared to $180,000 in 2021, according to a Reuters analysis. - Treatments for rare diseases now account for 72% of new drug launches, up from 51% in 2019, with over 40% of these orphan drug approvals targeting oncology indications. - The highest-priced new therapy is Orchard Therapeutics' Lenmeldy, a gene therapy for metachromatic leukodystrophy, costing patients over $4 million per year.
• XOMA Royalty doubled its portfolio to over 120 royalty assets through five strategic transactions in 2024, strengthening its position as a biotech royalty aggregator. • The company completed two whole company acquisitions and celebrated FDA approvals for Day One's OJEMDA™ (tovorafenib) and Zevra's MIPLYFFA™ (arimoclomol), generating significant milestone payments. • Despite reporting a net loss of $13.8 million for 2024, XOMA Royalty received $46.3 million in cash receipts and maintains over $100 million in cash, positioning it for sustainable cashflow from royalties.
- DelveInsight's latest report reveals 8+ companies developing 10+ therapies for hypersomnia, with Avadel's Lumryz, Zevra's KP1077, and Alkermes' ALKS 2680 among the key candidates advancing through clinical trials. - Recent clinical milestones include Axsome's AXS-12 meeting its Phase III primary endpoint with 72-82% reduction in cataplexy attacks, and Takeda's TAK-861 showing promise as the first treatment targeting the underlying pathophysiology of narcolepsy type 1. - The hypersomnia market is driven by increased awareness, unmet medical needs, and advances in neuroscience, though challenges remain including lack of biomarkers, high trial costs, and regulatory hurdles.
