Mastocytosis is a rare disease characterized by proliferation and accumulation of mast cells in various tissues, causing a wide variety of clinical symptoms. Indolent systemic mastocytosis, including the subvariant of smouldering systemic mastocytosis, is a lifelong condition associated with reduced quality-of-life.
Mastocytosis can be categorized into cutaneous mastocytosis and systemic mastocytosis. Systemic mastocytosis (SM) can be further categorized into indolent SM, smouldering SM and aggressive SM. Patients with indolent SM or smouldering SM have a (nearly) normal life expectancy, while patients with aggressive SM have a median overall survival of approximately 4 years.
There is a high unmet need in indolent systemic mastocytosis (ISM) and smouldering systemic mastocytosis (SSM) for new therapeutic options with demonstrated activity on severe symptoms and adequate safety profile for a life-long treatment. Masitinib is the leading program in ISM and a viable approach to this goal.