Company: ZVRA
Filing Date: 2025-05-08
Form Type: DEFA14A
Source: 0001193125-25-115961
Chunk: 6

Company: ZEVRA THERAPEUTICS, INC.
Filing Date: 2025-05-08
Form: DEFA14A
Chunk 6
---
 Treatment Adherence Remains in UCDs OLPRUVA UCDs are a group of rare inherited metabolic disorders resulting from a defect in one of the six enzymes or two transporters in the urea cycle; UCD causes an accumulation of ammonia, known as hyperammonemia, which can be toxic and lead to neurocognitive damage and death ~1,100 Individuals diagnosed in the U.S. ~80% Of patients have deficiencies in the CPS, OTC, or AS enzymesii >800 Patients in the U.S. are currently on alternative therapies for the treatment of certain UCDs >25% Of hyperammonemic crises from UCDs stem from poor treatment adherence to alternative therapies

Our first-ever commercially launched product helps people suffering from certain UCDs MIPLYFFA OLPRUVA U.S. Progress U.S. Progress Regulatory Path in EU EU-based Expanded Access Program continues with ~70 to 80 patients Target filing of Marketing Authorization Application (MAA) in Europe in 2H 2025 Strong launch of MIPLYFFA; made available to patients within 8 weeks following approval Received 109 prescription enrollments, as of December 31, 2024 Surpassed internal expectation of market access Enrolled all active U.S. Expanded Access Program participants Accelerating awareness and adoption of OLPRUVA; all Q1 enrollments align with our targeted patient segment Received 4 prescription enrollments, as of December 31, 2024 Increased market access for covered lives Refined strategy to target the adult-onset population, which is starting to show signs of uptake First approved product in the U.S. for the treatment of NPC Delivering Commercial Excellence Through Our Two Products Launched in 2024

MIPLYFFA Expanding adoption in U.S. and securing regulatory approval in the EU Launched educational and testing support for NPC providers in the U.S. Working with payors to secure patient access to treatment On track to file MAA in second half of 2025, potentially reaching 1,100 NPC patients in the EU OLPRUVA Expanding adoption Targeting adult-onset patients who benefit most from OLPRUVA’s portability and ease of use; assisting those with insurance challenges DiSCOVER trial Accelerating Phase 3 trial enrollment Accelerating Phase 3 trial enrollment for VEDS, a disease with no approved U.S. treatments affecting ~7,500 people As of Dec. 31, 2024, 27 patients enrolled in the DiSCOVER