Company: ZVRA
Filing Date: 2025-11-05
Form Type: 10-Q
Source: 0001434647-25-000011
Chunk: 189

Company: ZEVRA THERAPEUTICS, INC.
Filing Date: 2025-11-05
Form: 10-Q
Item: Part I, Item 2
Chunk 189
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 included the acquisition of OLPRUVA (sodium phenylbutyrate) for oral suspension, which was approved by the FDA on December 27, 2022, for the treatment of certain urea cycle disorders (UCDs). In addition, we acquired Acer's pipeline of investigational product candidates, including celiprolol for the treatment of Vascular Ehlers-Danlos syndrome (VEDS) in patients with a confirmed type III collagen (COL3A1) mutation.

In May 2022, we purchased all of the assets and operations of Orphazyme A/S (“Orphazyme”) related to arimoclomol and agreed to assume an estimated reserve clawback liability of $5.2 million related to revenue generated from the sale of arimoclomol for the treatment of NPC under the remunerated expanded access program in France, Accès Compassionnel (“French AC”).

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Table of Contents

Our Product Candidates and Approved Products

We have built a diverse portfolio of products and product candidates through a combination of internal development and strategic investments through acquisition. For example, we have employed our proprietary Ligand Activated Technology (LAT®) platform to develop approved products (e.g., AZSTARYS) and clinical development candidates (KP1077IH and KP1077N). Through our business development efforts, we have added commercial products (MIPLYFFA and OLPRUVA) and a clinical development candidate (celiprolol). We also have a variety of product candidates and compounds that are clinical-stage and designed to address a variety of rare diseases and other indications.

Current commercial products and active development assets are summarized in the table below:

Active Zevra Commercial and Active Development Assets

Parent Drug IndicationProduct / CandidateDevelopment StatusNext Milestone(s)ArimoclomolNiemann-Pickdisease type C(NPC)MIPLYFFAFDA ApprovedEuropean ExpandedAccess Program (“EAP”)Marketing Authorisation Application (“MAA”) Submitted to European Medicines Agency (“EMA”) July 2025Tracking U.S. CommercialProgressEMA DecisionSodium phenylbutyrateUrea CycleDisorders (UCD)OLPRUVAFDA ApprovedTracking CommercialProgressCeliprololVascular EhlersDanlos Syndrome(VEDS)CeliprololClinical - Phase 3Ongoing Phase 3 Trial Fully EnrolledSerdexmethylphenidateIdiopathicHypersomnia (IH)KP1077IHClinical