Givlaari est un m\u00e9dicament \u00e0 base de givosiran indiqu\u00e9 dans le traitement de la porphyrie h\u00e9patique aigu\u00eb (AHP) chez les adultes et les adolescents \u00e0 partir de 12 ans. La porphyrie h\u00e9patique aigu\u00eb est d\u00e9crite comme une maladie g\u00e9n\u00e9tique rare dans laquelle le foie ne parvient pas \u00e0 produire correctement l\u2019h\u00e8me, entra\u00eenant une accumulation de pr\u00e9curseurs responsables de crises caract\u00e9ris\u00e9es notamment par des douleurs abdominales s\u00e9v\u00e8res, des vomissements et des troubles neurologiques. En raison de la raret\u00e9 de cette pathologie, Givlaari a obtenu le statut de m\u00e9dicament orphelin le 29 ao\u00fbt 2016.<\/p>\n
Le m\u00e9dicament est administr\u00e9 par injection sous-cutan\u00e9e une fois par mois, \u00e0 une posologie ajust\u00e9e au poids du patient. Il n\u2019est disponible que sur prescription m\u00e9dicale et le traitement doit \u00eatre initi\u00e9 par un professionnel de sant\u00e9 exp\u00e9riment\u00e9 dans la prise en charge de cette maladie. Givosiran est un petit ARN interf\u00e9rent de synth\u00e8se qui r\u00e9duit la production d\u2019une enzyme intervenant pr\u00e9cocement dans la synth\u00e8se de l\u2019h\u00e8me au niveau h\u00e9patique, limitant ainsi l\u2019accumulation des substances \u00e0 l\u2019origine des sympt\u00f4mes.<\/p>\n
L\u2019Agence europ\u00e9enne des m\u00e9dicaments (EMA) a \u00e9valu\u00e9 un essai principal men\u00e9 chez 94 patients, montrant qu\u2019en moyenne les patients trait\u00e9s par Givlaari pr\u00e9sentaient trois crises s\u00e9v\u00e8res de porphyrie par an, contre treize pour ceux recevant un placebo. Les effets ind\u00e9sirables les plus fr\u00e9quents (plus d\u2019un patient sur cinq) sont les r\u00e9actions au site d\u2019injection, les naus\u00e9es et la fatigue. Les effets ind\u00e9sirables observ\u00e9s dans l\u2019\u00e9tude principale \u00e9taient le plus souvent d\u2019intensit\u00e9 l\u00e9g\u00e8re \u00e0 mod\u00e9r\u00e9e et se sont majoritairement r\u00e9solus au cours du suivi.<\/p>\n
Au vu de ce rapport b\u00e9n\u00e9fice\/risque jug\u00e9 favorable, l\u2019EMA a autoris\u00e9 la mise sur le march\u00e9 de Givlaari dans l\u2019ensemble de l\u2019Union europ\u00e9enne \u00e0 compter du 2 mars 2020. Le m\u00e9dicament fait l\u2019objet d\u2019une surveillance particuli\u00e8re avec un suivi continu des donn\u00e9es de s\u00e9curit\u00e9, et des recommandations sp\u00e9cifiques pour une utilisation s\u00fbre et efficace sont int\u00e9gr\u00e9es au r\u00e9sum\u00e9 des caract\u00e9ristiques du produit et \u00e0 la notice. Toute nouvelle proc\u00e9dure susceptible d\u2019affecter les informations sur le produit est r\u00e9f\u00e9renc\u00e9e, la derni\u00e8re mentionn\u00e9e \u00e9tant identifi\u00e9e par le code N\/000037727324\/09\/2026. (contenu r\u00e9sum\u00e9 en fran\u00e7ais par l’IA)\u00a0\u200bLire la Suite<\/a><\/p>","protected":false},"excerpt":{"rendered":" Givlaari est un m\u00e9dicament \u00e0 base de givosiran indiqu\u00e9 dans le traitement de la porphyrie h\u00e9patique aigu\u00eb (AHP) chez les adultes et les adolescents \u00e0 partir de 12 ans. 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