Myasthenia gravis, news in treatments: Aifa gives green light to Ravulizumab

A step forward in the fight against myasthenia gravis, a rare and chronic neuromuscular disease that affects approximately 17 thousand Italians and 89 thousand people throughout Europe.

Myasthenia gravis is characterized by muscle weakness that can vary in intensity throughout the day and often affects the face, neck, and limbs. Although its cause is still unclear, the disease is attributed to an attack by the immune system on muscle receptors, with symptoms ranging from drooping eyelids to difficulty breathing and swallowing. Diagnosis is complicated by the often subtle appearance of the symptoms, which can be mistaken for psychosomatic disorders.

The approval of ravulizumab, a long-acting complement C5 inhibitor, represents an important innovation for adult patients with generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive. This treatment will be an additional therapy to those already in use, and will be administered every eight weeks, for a total of six administrations per year, with clear benefits in terms of quality of life. “We are proud to make available in Italy a treatment that has not only proven effective, but can significantly improve the management of the disease”, said Anna Chiara Rossi, Vice President and General Manager of Alexion, AstraZeneca Rare Disease.

During the press conference held in Milan, Dr. Michelangelo Maestri Tassoni, an expert from the Department of Neuroscience at the Pisa University Hospital, explained the mechanisms of the disease. “The immune system attacks muscle receptors, interrupting movement signals and causing symptoms ranging from double vision to loss of balance, to compromising vital functions such as swallowing and breathing.”, the doctor said. Early diagnosis is essential to slow the progression of the disease and prevent complications that can be fatal.

Patient associations also welcomed this innovation. Mariangela Pino, representative of the Italian Association of Myasthenia and Immunogenerative Diseases (AIM) of Lazio, underlined the positive impact of a less frequent treatment: “The management of the disease is exhausting, with intensive treatments that often undermine the continuity of care. The dream of every myasthenic is to reach a dimension of normality, essential for their personal and professional fulfillment”.

Professor Renato Mantegazza, neurologist emeritus of the Carlo Besta Institute in Milan, highlighted the importance of ravulizumab as a step forward in the treatment of myasthenia gravis. “This disease severely affects the ability to carry out daily activities. The arrival of ravulizumab marks a significant step forward in improving the autonomy and serenity of patients”, said Mantegazza.

With the approval of ravulizumab, patients with myasthenia gravis will be able to count on a therapy that not only aims to control symptoms, but to reduce the impact of the disease on their daily lives. For AstraZeneca and Alexion, committed to research on rare diseases, this milestone represents an incentive to continue with patient support projects, going beyond the mere supply of drugs and responding to the still unmet needs of those who face the difficulties of myasthenia gravis every day.