• 2 months ago
(Adnkronos) - È ora disponibile e rimborsabile, dopo il via libera di Aifa, l’anticorpo monoclonale ravulizumab, sviluppato da Alexion - AstraZeneca Rare Disease per il trattamento del disturbo dello spettro della neuromielite ottica (Nmosd) in adulti positivi all'anticorpo anti-aquaporina-4 (AQP4) (Ab+), malattia autoimmune rara che in Italia interessa circa 1500-2000 persone.Sicuro ed efficace nel ridurre le ricadute nella quasi totalità dei casi, come dimostrato dallo studio di fase 3 Champion - Nmosd, ravulizumab è il primo inibitore del complemento C5 a lunga durata d’azione per il trattamento di questa malattia rara.

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00:00There are about 1,500-2,000 people, mostly women, who in Italy suffer from Neuromyelitheotic
00:10Spectrum Disorder, a rare autoimmune disease in which the immune system attacks tissues
00:16and healthy cells of the central nervous system.
00:19The relapses of the disease, unpredictable and characterized by neurological symptoms,
00:23can cause cumulative disability and thus compromise the quality of life of patients.
00:28For adults affected by Neuromyelitheotic Spectrum Disorder positive to the antibody Anti-Aquaporin-4
00:34the monoclonal antibody Ravulizumab is now available and refundable.
00:38Developed by Alexion AstraZeneca Rare Disease,
00:41it is the first inhibitor of the long-term action complement C5 for the treatment of this rare disease.
00:47This drug has shown to be effective, I must say, never seen in previous studies.
00:53I had the good fortune to experiment on patients with this drug
00:57and to be able to see that none of the patients studied in the trial
01:01in the period of almost two years have been relapsed.
01:05Now we also have a greater follow-up, which is about two and a half years.
01:10The data were recently presented at a conference in Canada.
01:14And I must say that this effectiveness has been maintained over time.
01:19A drug that is supplied by endovenose every two months,
01:26thus also facilitating a little the convenience in terms of the patient
01:31compared to similar drugs that were injected every 15 days.
01:37The free route of AIFA, the refundability of Ravulizumab is important,
01:40but it is equally essential to guarantee equal access to the cures to all patients.
01:46It is important that there are reference centers,
01:49because people must be able to find in their territorial reality
01:54who is able to do an accurate tempestuous diagnosis.
01:59Diagnosis that does not stop as a first step,
02:04because in reality you need a monitoring, a cure that must continue
02:10up to the evaluations through the center, through the territorial network
02:16of all those needs that must find answers to give the person the possibility
02:21to live their own life in addition to the disease.
02:24Alexio's commitment to research and development of transformative drugs
02:27for people affected by rare diseases has lasted for over 30 years.
02:31With this line of research we have managed to make available
02:36to people affected by neuromyelitis, which is a very serious disease
02:41and highly disabling, a very effective solution
02:46that allows them to live an equally excellent quality of life.

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