Italy has approved crovalimab, a first-in-class monoclonal antibody, as a treatment for paroxysmal nocturnal hemoglobinuria (PNH), a rare and chronic blood disorder. The approval, granted by the Italian Medicines Agency (AIFA) on February 17, 2026, marks the availability of the first subcutaneous treatment for PNH that can be self-administered at home, according to a statement released by PharmaStar.
Crovalimab is indicated for both treatment-naïve and previously treated adult and adolescent patients (12 years and older, weighing at least 40 kg) with PNH. Currently, treatment options often require frequent intravenous infusions and hospital visits. The new subcutaneous formulation offers a significant shift in patient care, potentially improving quality of life and adherence to therapy.
PNH is caused by an acquired genetic mutation in hematopoietic stem cells, leading to the production of red blood cells lacking protective proteins. This makes them susceptible to destruction, resulting in anemia, fatigue, thrombosis, kidney damage, and a substantial impact on overall well-being. Bruno Fattizzo, a hematologist and researcher at the University of Milan, explained that PNH affects an estimated 10 to 20 people per million, translating to approximately 1,000 cases in Italy. Without effective treatment, the prognosis is severe, with a 35% mortality rate within five years and a median survival of around 10 years from diagnosis, based on historical data.
The symptoms of PNH are often non-specific, including intense fatigue, shortness of breath, abdominal pain, and headaches, making diagnosis challenging. While C5 inhibitors have significantly improved survival rates and reduced hemolysis and thrombotic events, they necessitate intravenous administration. Crovalimab offers a new approach with its monthly subcutaneous injection.
Annalisa Lazzini, President of AIEPN – the Italian Association for Paroxysmal Nocturnal Hemoglobinuria – emphasized the importance of continued collaboration between patient associations, institutions, the clinical community, and pharmaceutical companies to ensure sustainable and concrete responses to the needs of individuals living with PNH.