- In January 2025, Eton Pharmaceuticals announced the acquisition of Galzin (zinc acetate), a drug approved by the FDA for the maintenance treatment of Wilson’s disease. This acquisition underscores Eton's commitment to addressing rare diseases and enhancing patient access to essential therapies. The company plans to commence marketing Galzin in the U.S. in the first quarter of 2025
- In October 2024, Monopar Therapeutics announced an exclusive global licensing agreement with Alexion, AstraZeneca Rare Disease, for ALXN-1840 (bis-choline tetrathiomolybdate), a Phase 3 drug candidate for Wilson’s disease. Monopar will oversee all future development and commercialization efforts, aiming to bring this promising therapy to patients worldwide
- In October 2024, Ultragenyx Pharmaceutical reported encouraging outcomes from Stage 1 of the Phase 1/2/3 Cyprus2+ study of UX701, an investigational gene therapy for Wilson’s disease. The therapy demonstrated significant clinical activity and improved copper metabolism, with several patients discontinuing standard-of-care treatments across all three dose cohorts. A new cohort with a higher dose and optimized immunomodulation will be added to enhance efficacy.
- In June 2024, Vivet Therapeutics presented interim results from its Phase 1/2 GATEWAY trial at the EASL Congress 2024 in Milan, Italy. The trial is evaluating the safety, pharmacodynamics, and effectiveness of VTX-801, Vivet’s leading treatment candidate for Wilson’s disease. These findings highlight the potential of VTX-801 as a novel therapeutic option for patients
- In September 2023, UC Davis Health researchers administered the first-ever gene therapy for a patient with Wilson’s disease as part of the CYPRUS2+ clinical trial. The trial displayed positive results for UX701, an investigational gene therapy treatment, marking a significant milestone in the treatment of this rare genetic disorder
