Rare diseases, Aifa ok to therapy for neuromyelitis optica spectrum disorder

The Italian Medicines Agency (AIFA) has approved the reimbursement of ravulizumab for the treatment of adults with neuromyelitis optica spectrum disorder (NMOSD) who are positive for the anti-aquaporin-4 (AQP4) antibody (Ab+).They announce it in a note, AstraZenecatogether with Alexion, AstraZeneca Rare Diseasespecifying that ravulizumab is the first long-acting complement C5 inhibitor for the treatment of this rare disease. The reimbursement of ravulizumab by Aifa, which follows the approval by the European Commission in May 2023, is based on the positive results from the Phase 3 Champion-Nmosd study, during which no relapses of the disease were reported in patients treated with ravulizumab with a median treatment duration of 73 weeks and continuing with a median duration of 90 weeks.

NMOSD is a rare and debilitating autoimmune disease that affects the central nervous system, particularly the spinal cord and optic nerves. Most people who are affected experience unpredictable relapses, characterized by a new onset of neurological symptoms or a worsening of existing symptoms, which tend to be severe and recurrent and can cause permanent disability. It is estimated that in Italy there are 1,500-2,000 people living with this disease. “The optimal management of Nmosd – explains Eleanor Cocco, full professor of Neurology at the University of Cagliari and director of the Regional Multiple Sclerosis Unit, ASL Cagliari/University of Cagliari – first of all implies avoiding the onset of relapses, which are characterized by a new onset of neurological symptoms or a worsening of existing ones, which tend to be serious and recurrent: even a single relapse can cause irreversible disabilities, determining a reduction in the autonomy and quality of life of those affected”.

As he observes Charles PozzilliFull Professor at the Department of Human Neurosciences, La Sapienza University and Director of the Multiple Sclerosis Center, S. Andrea Hospital in Rome, “the approval of ravulizumab by AIFA provides Italian patients with NMOSD and their doctors with an innovative, long-acting treatment that can reduce the risk of relapses to a degree never achieved before. The phase 3 study has in fact highlighted the absence of relapses for an average of 73 weeks with administration of the drug every eight weeks. An important result that offers patients a new therapeutic option for managing the disease”.

The announcement of the reimbursement of ravulizumab in Italy was welcomed by Aism (Italian Multiple Sclerosis Association) and from Let’s go (Italian Association of Optic Neuromyelitis), the associations that represent patients. “This is excellent news for people in Italy who suffer from Optic Neuromyelitis, a disease that is still incurable but that today, thanks to the progress of medical-scientific research, can be faced with greater confidence” – he underlines Mario Alberto Battagliafounding member and Vice President of Ainmo and general manager of Aism – The commitment must now continue, indeed, intensify, on the treatment front by making available in the Regions and in the Health Authorities with the National Health Service the best drug for the clinical condition of each person, as well as on that of the correctness and timeliness of the diagnosis in the recognized reference Clinical Centers and of the support to patients in all health and social areas”.

Neuromyelitis optica spectrum disorder “is an insidious disease that is difficult to diagnose and has an unpredictable course,” he reiterates. Anna Chiara RossiVP & General Manager Italy, Alexion, AstraZeneca Rare Disease – which unfortunately can have a very serious impact on the lives of those who suffer from it. The approval of the reimbursement of ravulizumab in Italy represents a significant step forward in the treatment of this rare disease, an innovative and effective therapeutic solution that has proven to be able to truly change the lives of patients. We are proud of this result, the fruit of Alexion’s constant commitment to provide transformative therapies and improve the lives of people with rare diseases and their loved ones”.

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