Zevra Therapeutics announced the presentation of long-term real-world data on MIPLYFFA (arimoclomol) for treating Niemann-Pick disease type C at the 2026 SSIEM Annual Symposium. Data from U.S. and German early access programs demonstrate sustained disease stabilization over up to 8 years with a consistent safety profile across diverse patient populations, including adults and children. The pivotal trial showed a 65% reduction in disease progression compared to placebo.

Zevra Therapeutics announced that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) issued a negative opinion on the Marketing Authorization Application for arimoclomol to treat Niemann-Pick disease type C. The company plans to request a re-examination of the decision and continues supporting patient access through its Expanded Access Program.

XOMA Royalty Corporation reported over $50 million in cash receipts for 2025, with royalties up 68% year-over-year. The company added 22 assets to its portfolio, completed seven acquisitions generating $11.7 million in non-dilutive capital, and repurchased 648,048 shares for $16 million. Key 2026 catalysts include Phase 2b/3 data readouts and potential EMA regulatory decisions on commercial assets OJEMDA and MIPLYFFA.

Zevra Therapeutics announced granting options to purchase 38,000 shares of common stock to two new employees under its 2023 Employment Inducement Award Plan, with options vesting over four years.

Zevra Therapeutics has entered into an agreement to sell its Rare Pediatric Disease Priority Review Voucher for $150 million, strengthening its balance sheet and supporting its strategic priorities.

Zevra Therapeutics is consolidating its development and scientific functions under the Chief Medical Officer, resulting in the departure of the Chief Development Officer and Chief Scientific Officer. The company is also discontinuing its in-house drug discovery activities and closing its laboratory facilities to focus on late-stage clinical development and commercial opportunities.

Zevra Therapeutics announced the commercial availability of MIPLYFFA, the first FDA-approved treatment for Niemann-Pick disease type C. The company's comprehensive patient support program, AmplifyAssist, is in place to address access barriers.

Zevra Therapeutics will present data at the 53rd Child Neurology Society Annual Meeting, demonstrating the relevance of the swallow domain in the Niemann-Pick disease Type C Clinical Severity Scale (NPCCSS). The study indicates that the NPCCSS swallow score reflects the patient's level of dysfunction, and a change in score reflects actual improvement or worsening in a patient's swallowing function.

The FDA Genetic Metabolic Diseases Advisory Committee voted favorably that the data support arimoclomol as an effective treatment for Niemann-Pick disease type C, a rare and progressive neurodegenerative disorder. The committee's recommendation will be considered by the FDA as it completes its review of the arimoclomol new drug application.

Rahsaan W. Thompson appointed as Chief Legal Officer, Secretary and Compliance Officer

CELEBRATION, Fla. and ELK GROVE VILLAGE, Ill., June 18, 2024 (GLOBE NEWSWIRE) -- Orsini Specialty Pharmacy (Orsini), and Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra) today announced that Orsini is now the pharmacy partner for OLPRUVA® (sodium phenylbutyrate) for oral suspension. OLPRUVA® is a prescription medicine used along with certain therapies, including changes in diet, for long-term management of certain adult and pediatric patients with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC) or argininosuccinic acid synthetase (AS). Visit OLPRUVA-Prescribing-Information.pdf (olpruva.com) to view the full Prescribing Information, including Important Safety Information.

KP1077 was well tolerated and showed meaningful clinical improvements in patient-reported assessments of daytime sleepiness, sleep inertia, and brain fog