- In March 2025, Cycle Pharmaceuticals Ltd. announced the launch of VENXXIVA™ (tiopronin) delayed-release tablets in the U.S. for patients with severe homozygous cystinuria unresponsive to standard measures. This FDA-approved treatment is available in 100 mg and 300 mg doses and is indicated for the prevention of cystine stone formation in adults and pediatric patients aged 9 years and older. A 2020 study demonstrated that patients on tiopronin therapy reported improved health-related quality of life, including better scores in social, emotional, disease, and vitality impact
- In February 2025, Advicenne received Orphan Drug Designation (ODD) from the U.S. FDA for ADV7103, a novel formulation of potassium sodium hydrogen citrate for cystinuria treatment. This designation aims to expedite the development and regulatory review of ADV7103, potentially enhancing treatment options for patients with cystinuria
- In January 2024, Travere Therapeutics announced positive top-line results from its Phase 3 clinical trial of sparsentan for the treatment of adults with cystinuria. Sparsentan is an orally administered dual endothelin receptor antagonist, and these results support its potential as a therapeutic option for cystinuria patient
- In May 2024, AstraZeneca initiated a Phase 2 clinical trial of its investigational drug, AZD9829, for the treatment of cystinuria. AZD9829 is a novel small molecule inhibitor of cystine transport, and this trial aims to evaluate its safety and efficacy in cystinuria patients
- In July 2024, Pfizer Inc. announced the acquisition of Arena Pharmaceuticals, a biopharmaceutical company with a focus on developing therapies for inflammatory diseases. This acquisition expands Pfizer's portfolio in the area of rare diseases, including cystinuria, potentially accelerating the development of new treatment options for patients
