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Italy has approved a first-of-its-kind treatment for paroxysmal nocturnal hemoglobinuria (PNH), a rare and chronic blood disorder. The fresh therapy, Crovalimab, developed by Roche, offers a monthly subcutaneous injection that can be self-administered at home, potentially improving the quality of life for those affected by this challenging condition. The approval, granted by AIFA (Agenzia Italiana del Farmaco), marks a significant advancement in PNH treatment options.
PNH is caused by an acquired genetic mutation in blood stem cells, leading to the production of red blood cells vulnerable to premature destruction. This destruction occurs both within blood vessels and in the liver and spleen, resulting in anemia, significant fatigue, increased risk of blood clots, kidney damage and a substantial impact on overall well-being. According to Dr. Bruno Fattizzo, a hematologist and researcher at the University of Milan, approximately 10 to 20 people per million are affected by PNH, translating to roughly 1,000 individuals currently living with the disease in Italy [SaluteH24]. Without effective treatment, the prognosis can be severe, with a 35% mortality rate within five years and a median survival of approximately 10 years from diagnosis in historical cohorts.
What is Crovalimab and How Does it Work?
Crovalimab is a monoclonal antibody designed to block the complement system, a part of the immune system that contributes to the destruction of red blood cells in PNH patients. Specifically, it targets C5, a key protein in the complement cascade, inhibiting its activity and providing rapid and sustained protection against red blood cell breakdown [SaluteH24]. The treatment is approved for both adults and adolescents (aged 12 and above, weighing at least 40 kg) who are new to treatment or have previously been treated with C5 inhibitors.
A New Approach to PNH Management
The approval of Crovalimab represents a shift towards more convenient and patient-centric PNH management. The subcutaneous administration route, coupled with the monthly dosing schedule, allows for self-administration at home after an initial period under medical supervision. This contrasts with some existing treatments that require frequent intravenous infusions in a clinical setting [VogliadiSalute]. The aim is to not only offer new therapeutic perspectives but also to enhance the patient experience and improve their overall quality of life.
Recent Pharmaceutical Approvals in Italy
The approval of Crovalimab is one of several recent pharmaceutical advancements in Italy. On February 17, 2026, it was announced that 36 new drugs had come into commerce, alongside 13 commercializations being ceased and 3 price variations [Pharmastar]. The AIFA recently approved olaparib for specific types of BRCA-mutated pancreatic cancer patients and serplulimab for minor cell lung cancer, demonstrating a period of increased activity in the approval of novel therapies.
The introduction of Crovalimab offers a significant step forward in the treatment of PNH, providing a new option for patients and potentially improving their long-term outcomes. Further monitoring and real-world data collection will be crucial to fully assess the long-term benefits and impact of this innovative therapy.
Disclaimer: This article provides informational content and should not be considered medical advice. Always consult with a qualified healthcare professional for diagnosis and treatment of any medical condition.
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