Company: ZVRA
Filing Date: 2025-11-05
Form Type: 10-Q
Source: 0001434647-25-000011
Chunk: 88

Company: ZEVRA THERAPEUTICS, INC.
Filing Date: 2025-11-05
Form: 10-Q
Item: Part I, Item 1
Chunk 88
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.S. CommercialProgressEMA DecisionSodium phenylbutyrateUrea CycleDisorders (UCD)OLPRUVAFDA ApprovedTracking CommercialProgressCeliprololVascular EhlersDanlos Syndrome(VEDS)CeliprololClinical - Phase 3Ongoing Phase 3 Trial Fully EnrolledSerdexmethylphenidateIdiopathicHypersomnia (IH)KP1077IHClinical - Phase 2Phase 3 Trial Ready; Seeking Strategic AlternativesSerdexmethylphenidateNarcolepsyKP1077NClinical - Phase 1/2Phase 3 Trial Potential; Seeking Strategic AlternativesSerdexmethylphenidateand dexmethylphenidateAttention Deficitand HyperactivityDisorder (ADHD)AZSTARYSFDA Approved andPartneredCollecting Royalties andMilestones

These anticipated milestones are based on information currently available to us. Our current plans and expectations are subject to a number of uncertainties, risks and other important factors that could materially impact our plans, including risks which are not solely within our control. See Part I, Item 1A. “Risk Factors” of our Annual Report on Form 10-K for the fiscal year ended December 31, 2024, filed with the SEC on March 12, 2025, as updated by Part II, Item 1A. “Risk Factors” of this Quarterly Report on Form 10-Q.

MIPLYFFA

NPC is characterized by an inability of the body to transport cholesterol and lipids inside of cells. Symptoms of NPC include a progressive impairment of mobility, cognition, speech, and swallowing, often culminating in premature death. The incidence of NPC is estimated to be one in 100,000 to 130,000 live births. We estimate that there are approximately 2,000 individuals with NPC in the U.S. and Europe combined, of which approximately 900 are in the U.S. and 1,100 are in Europe, where there is a mature market with an approved treatment available for NPC. Of this estimated population, approximately 300 to 350 people have been diagnosed in the U.S. However, low diagnostic rates may affect the number of potential patients, and we believe that the availability of treatment options in the U.S. could increase awareness of the disease and assist in more accurately identifying patients. 

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On September 20, 2024, the FDA approved the NDA for MIPLYFFA