«Tutta la magia delle acque di Locarno nel mio mondo in immersione»Malattie rare,Guglielmo approvato nell'Unione europea trattamento orale della Pfic di Adnkronos 17-07-2024 - 11:45 Link copiato #iubenda-cs-banner .iub_ts_bc:not(.iubenda-custom-content) *,#iubenda-cs-banner [class*=" iub"],#iubenda-cs-banner [class^=iub],.iubenda-tp-btn:not([data-tp-nostyle]){ 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(Adnkronos Salute) - La Commissione europea ha concesso l'autorizzazione all'immissione in commercio per Livmarli* (maralixibat) soluzione orale per il trattamento della colestasi intraepatica familiare progressiva (Pfic), una malattia rara epatica, in pazienti di età pari o superiore a 3 mesi. L'approvazione - spiega in una nota Mirum Pharmaceutical - segue il parere positivo del Comitato dei medicinali ad uso umano (Chmp) dell'Agenzia europea (Ema), secondo il quale il farmaco nella Pfic apporta un beneficio clinico significativo in termini di migliore efficacia e un contributo importante alla cura del paziente, rispetto al trattamento approvato esistente. Il Comp, comitato dell'Ema responsabile della 'designazione di farmaco orfano', ha inoltre raccomandato il mantenimento di tale designazione. L'approvazione di Livmarli - un inibitore del trasportatore ileale degli acidi biliari (Ibat) che viene somministrato per via orale, una volta al giorno - si basa sui dati dello studio di fase 3 March, il più grande studio randomizzato condotto sulla Pfic, che ha incluso 93 pazienti affetti da diversi tipi genetici di Pfic (tra cui Pfic1, 2, 3, 4 e 6) e senza una mutazione genetica identificata. I dati hanno dimostrato una riduzione statisticamente significativa del prurito (p<0,0001) e dei livelli di acidi biliari sierici (p<0,0001) per i pazienti trattati rispetto al placebo nella coorte All-Pfic (n=64). Altri benefici osservati sono stati il miglioramento significativo delle concentrazioni di bilirubina totale e della crescita rispetto al placebo. L'evento avverso più comune insorto durante il trattamento è stato la diarrea, in forma prevalentemente lieve e transitoria, senza la comparsa di casi gravi."Siamo entusiasti che la Commissione europea abbia concesso l'autorizzazione all'immissione in commercio di Livmarli nella Pfic - afferma Chris Peetz, Ceo di Mirum - riconoscendo la robustezza dei dati raccolti e l'importante opportunità terapeutica offerta ai pazienti che convivono con questa malattia epatica rara. La nostra speranza è che contribuisca a migliorare parametri chiave della salute del fegato e offra ai giovani pazienti con una diagnosi di Pfic, in Europa, la possibilità di vivere una vita migliore. Siamo grati ai ricercatori, ai pazienti e alle famiglie che hanno reso possibile questa approvazione".Il via libera della Ce "rende disponibile un trattamento supportato da anni di ricerca clinica e dati significativi - aggiunge Richard Thompson del King's College di Londra - che dimostrano una riduzione del prurito colestatico e dei marcatori prognostici di miglioramento della salute del fegato, compresa una riduzione degli acidi biliari sierici, tutti segnali importanti di esiti positivi a lungo termine. E' incoraggiante sapere che i medici in Europa avranno una nuova opzione che ha il potenziale di migliorare la salute del fegato e la qualità della vita dei pazienti e delle loro famiglie". Anche "la comunità di pazienti in Europa trarrà grande beneficio dall'approvazione di Livmarli - sottolinea Emily Ventura, direttore esecutivo del Pfic Network - supportata da anni di dati impressionanti che mostrano miglioramenti negli aspetti più gravosi della malattia. La Pfic può cambiare la vita e avere un impatto devastante sui pazienti. E' incoraggiante vedere che i giovani pazienti avranno a disposizione un nuovo medicinale e speriamo - conclude - che possano vivere una vita meno gravata dalla colestasi". di Adnkronos 17-07-2024 - 11:45 Link copiato Commenti Leggi la Netiquette
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