(Adnkronos) - È disponibile in Italia ravulizumab inibitore del complemento C5 a lunga durata d'azione, come terapia aggiuntiva a quella standard per il trattamento di pazienti adulti affetti da Miastenia Gravis generalizzata (gMG - generalised Myasthenia Gravis) e positivi agli anticorpi anti-recettore dell’acetilcolina (AChR). L’annuncio del via libera al rimborso da parte dell’Agenzia del farmaco (Aifa), è stato dato da AstraZeneca, insieme ad Alexion, AstraZeneca Rare, durante un incontro con la stampa, a Milano.
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00:00AstraZeneca, together with Alexion AstraZeneca Rare Disease, announced that the Italian
00:10Pharmaceutical Agency has approved the reimbursement of Ravulizumab, inhibitor of the long-term
00:16C5 complement, as an additional therapy to the standard therapy for treating adult patients
00:22affected by generalized serious myasthenia.
00:26The topic was discussed at the meeting of a new management of generalized serious myasthenia
00:31approved Ravulizumab for the treatment of adult patients, which was held at Terrazza
00:36Gym in Milan.
00:37Ravulizumab is a monoclonal antibody, humanized, specific for the C5 fragment of the complement.
00:43The complement has a fundamental role in serious myasthenia, as it is activated by specific
00:48autoantibodies for the receptor of acetylcholine and the activation of the complement has
00:54the consequences of etherea, as it alters the morphology of neuromuscular junction, thus preventing
00:59the transmission of the impulse between the nerve and the muscle, causing the symptoms of the
01:03pathology.
01:04Generalized serious myasthenia is a pathology with a strong impact on patients and this new
01:10therapy can significantly improve the quality of life.
01:13The arrival of this new therapy is extremely important, the indications for use are those
01:21as an addition to the normal therapy, but what happens is that it quickly improves the
01:29symptoms, because refractory patients are the first line to be involved and above all
01:41it allows to significantly reduce conventional therapies, which are those that are more
01:49exuberant of side effects, in particular steroidal therapy.
01:57Once again, Alexion's commitment to transgenic rare disease is confirmed when dealing with
02:03rare diseases, combining research, innovation and patient care.
02:06We believe that it is essential not only to deal with research and development of innovative
02:14therapies, but also to collaborate with the whole ecosystem that runs and is part of the
02:19world of rare diseases to try to improve the daily life of these patients.
02:23This is because the path of the rare patient is a path of obstacles, starting with diagnosis,
02:29then of course the possibility of having an effective therapy available and then also in
02:34living what is the daily life.
02:38In doing this, we believe that it is essential to work in concert with the Association of
02:45Patients, with the scientific community and also with the institutions to really understand
02:50what the needs are and to create solutions together, because only in this way can we
02:55really be able to create something useful.
02:59And this is our way of working, what we do every day.