Summary
Approximately 1,600 prevalent cases were diagnosed across the 7MM in 2025, underscoring the condition's rarity and the concentration of patients at specialized metabolic and nephrology centers.
Treatment is limited to long-standing cysteamine products, PROCYSBI, CYSTAGON/NICYSTAGON (cysteamine bitartrate), and CYSTADROPS/CYSTARAN (cysteamine hydrochloride). Persistent tolerability issues, high dosing burden, and adherence challenges create a clear unmet need for better therapeutic options.
Development is progressing with emerging therapies, including TTI-0102, DFT383, and others. This focused pipeline reflects gradual yet meaningful innovation beyond...
