The drug is approved on a reimbursement basis for adult patients positive for anti-acetylcholine receptor antibodies
The Italian Medicines Agency (AIFA) has approved the reimbursement of the drug ravulizumab for the treatment, in addition to standard therapy, of adult patients suffering from generalized myasthenia gravis (gMG) and positive for anti-acetylcholine receptor (AChR) antibodies. gMG is a rare, debilitating, chronic autoimmune neuromuscular disease that leads to loss of muscle function and severe weakness. In Italy there are an estimated 17,000 people affected by the disease.
“In myasthenia gravis, the immune system attacks a receptor in the muscles, resulting in disrupted movement signals”, explains the Dr. Michelangelo Maestri TassoniDepartment of Neurosciences, Pisa University Hospital. “This leads to mild but still disabling symptoms, such as double vision and loss of balance, up to more serious conditions, which involve impaired swallowing and breathing. It is a disease that in women most often begins before the age of 40, with a significant impact on the quality of life.”
AIFA’s decision to authorize the reimbursement of ravulizumab for gMG follows the approval of the drug by the European Commission in September 2022, in turn based on the results of the Phase III CHAMPION-MG clinical trialpublished online in the magazine NEJM Records. In experimentation, ravulizumab was superior to placebo in the primary endpoint of change in Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total scorean index that measures the patient’s ability to carry out daily activities.
“Ravulizumab acts specifically on the C5 protein of the complement system, blocking its action and preventing the destruction of the neuromuscular junctionthus reducing the symptoms of weakness and fatigue”, specifies the Dr. Raffaele IorioCatholic University of the Sacred Heart, UOC Neurology Fondazione Policlinico Universitario A. Gemelli IRCCS. “AIFA’s approval of ravulizumab represents a notable step forward for the treatment of generalized myasthenia gravis in our country, as it provides patients and the doctors who treat them with a long-lasting therapeutic option. action capable of bringing lasting improvements in daily life. In fact, the drug has demonstrated clinical benefit in a wide range of patients, including those who continue to present symptoms of the disease even after initial treatment with standard therapy.”
The opinion of theItalian Association of Myasthenia and Immunodegenerative Diseases Amici del Besta (AIM)to which patients suffering from myasthenia gravis refer. The president of AIM, Professor Renato Mantegazzacommented on the AIFA decision as follows: “This disease has a debilitating impact on quality of life and severely affects the patient’s ability to carry out normal everyday activities. It is therefore to be welcomed with satisfaction the arrival of a treatment that has demonstrated that it can intervene effectively on these aspects”.
Mariangela Pino, secretary and member of AIM Lazio sectiondraws attention to a particularly important aspect for patients: “The management of the disease is decidedly very tiring due to the intensity and frequency of treatments, and this can often lead to poor adherence to therapy. In the case of ravulizumab, administration every 8 weeks represents an important therapeutic innovation for us patients and a crucial turning point that allows us to face everyday life differently. All this means being able to aspire to a dimension of ‘normality’ which is fundamental for the personal, social and professional fulfillment of each one.”
“We are proud to make available to Italian patients a treatment for generalized myasthenia gravis that proves to be effective and can simplify the management of the pathology, helping to change the lives of those affected and their families for the better,” underlines Anna Chiara RossiVP & General Manager Italy, Alexion, AstraZeneca Rare Disease. “It is an important result for us, but our commitment is not limited to the research and development of new drugs. We want to be a partner alongside the scientific community, patient associations, institutions and all those who work within the rare disease system, and act in a concrete way not only by making innovative therapeutic solutions available, but with projects ‘beyond the drug’ to respond to the still unmet needs of patients with rare diseases”.
