MILAN (ITALPRESS) – The Italian Pharmaco Agency (AIFA) has admitted to the Iptacopan Refundability, the first oral inhibitor of Factor B of the alternative supplement, first in its class, for the treatment of adults with glomerulopathy from C3. This result, together with the recognition of the status of innovation, responds to a dissatisfied therapeutic need for patients with C3G being this the first therapy authorized and reimbursed with a mechanism of action specific for the evolution of the disease or the disregulation of the alternative pathway of the central complement in the pathogenesis of the C3G. This approach allows action on the cause of disease and not only on symptoms, with a potential benefit in terms of slowing renal progression, stability of eGFR and reduction of proteinuria. Glomerulopathy from C3 is an ultra-rare and progressive kidney disease, with an Italian prevalence of 1-1,5 cases every 1,000,000 people. The disease may have an early onset in pediatric age, but is often diagnosed at the age of young adult, and has a severe prognosis: within ten years of diagnosis about half of patients can develop advanced kidney failure, making it necessary to resort to dialysis or kidney transplant, with a significant impact on personal, working and social life. “The diagnosis of C3G requires an in-depth specialist framework and is based on renal biopsy, currently the gold diagnostic standard – comments Professor Giuseppe Grandaliano, Ordinary of Nephology at the Catholic University of the Sacred Heart, seat of Rome and Director of the UOC of Nephrology of Fondazione Policlinico Gemelli IRCCS – An accurate diagnosis is fundamental not only to confirm pathology, but also to guide the path. The role of the complement and the mechanism of action of the molecule. The complement is a component of the immune system, consisting of a set of proteins that contribute to the defense of the body. In the C3G, this system is activated abnormally and persistently, causing the accumulation of C3 protein in glomeruli and progressive damage to the kidney. Within this mechanism, Factor B plays a key role as a “changer” of the alternative complement path. The molecule acts by selectively blocking Factor B, reducing the uncontrolled activation of the complement and helping to protect the glomeruli from inflammatory damage. In the study of Phase III APPEAR-C3G, at the basis of AIFA approval, the molecule showed a reduction of proteinuria of 35.1% compared to the placebo, with a favorable profile of safety and tolerability; the reduction of proteinuria accompanied a stabilization of the kidney function (eGFR) in the follow-up. Nefrologia Dialisi Trapianto U, Presidio Molinette e CTO – AOU Città della Salute e della Scienza di Torino – With this approval a significant therapeutic vacuum is filled: the availability of a tool that selectively intervenes on the cause at the base of the disease represents a significant change in the treatment landscape”. C3G may already occur in paediatric age, but is often diagnosed later in time, with an average age diagnosed around 21 years and with a significant impact on daily life limiting school, work and social activity; about 25% of patients report school or work absences and 21% suffer from disease-related depression. “For those who live with C3G, the difficulty is not only clinical: the path leading to diagnosis is often long and complex, anthat because it is a pathology still little known. Once diagnosed, many patients struggle to identify a stable clinical reference and are forced to move away from their region, with a significant impact on everyday life and family organization – says Fabrizio Spoleti, President of the patient association DDD ETS Project – Patients report the need for more accessible information, clearly identifiable reference centers and more coordinated care paths. Disease management requires frequent monitoring and time spent in the hospital, with relapses on school, work and quality of life. As an association, we work to break this isolation, foster knowledge sharing and help build ever more clear networks and care addresses, even through a collaboration between centers at national and international level.” Thanks to a history of over 40 years of transplantation, Novartis’ commitment to research and development is aimed at protecting the kidney function and delaying or preventing dialysis and/or transplantation. In addition to the commitment in the C3G, the pipeline also includes other unsatisfied high-demand kidney pathologies, such as IgA (IgAN) nephropathy and further glomerular conditions under clinical study. “Admission to repayment of this molecule is an important milestone for Italian patients with C3G – concludes Paola Coco, Country Chief Scientific Officer and Head of Medical Affairs of Novartis Italy – This result confirms our commitment to developing targeted solutions for complex and unsatisfied clinical pathologies. The recognition of innovativeness attributed to the molecule further enhances the contribution of therapeutic innovation in this context.” -photo f12/Italpress – (ITALPRESS).
