I-Rezdiffra (resmetirom) ivunyiwe yi-FDA yase-USA kunyango lwabantu abadala abane-noncirrhotic non-alcoholic steatohepatitis (NASH) ene-moderate to advanced yesibindi scarring (fibrosis), ukuba isetyenziswe kunye nokutya kunye nokuzilolonga.
Ukuza kuthi ga ngoku, izigulana ezine-noncirrhotic non-alcoholic steatohepatitis (NASH) nazo ezinezilonda eziphawulekayo zesibindi bezingenalo iyeza elinokujongana ngqo nazo. ukulimala kwesibindi. Ii-FDA's Ukwamkelwa kweRezdiffra kuya kuthi, okokuqala, kubonelele a impatho ukhetho kwezi zigulana, ukongeza ukutya kunye nokuzilolonga.
I-NASH isiphumo sokuqhubela phambili kwamafutha angenalo utywala isibindi isifo apho isibindi ukudumba, ekuhambeni kwexesha, kunokukhokelela kwizilonda zesibindi kunye nokungasebenzi kakuhle kwesibindi. I-NASH ihlala ihambelana nezinye iingxaki zempilo ezifana noxinzelelo lwegazi kunye nohlobo lwe-2 yeswekile. Ngoqikelelo olunye ubuncinane, malunga ne-6-8 yezigidi zabantu e-US bane-NASH ephakathi ukuya kwinqanaba eliphezulu lokukrala kwesibindi, kunye nelo nani kulindeleke ukuba lonyuke.
I-Rezdiffra yi-activator inxalenye ye-receptor yehomoni ye-thyroid; ukusebenza kwale receptor nguRezdiffra kwisibindi kunciphisa ukuqokelela kwamafutha esibindi.
Ukhuseleko kunye nokusebenza kakuhle kweRezdiffra
Ukhuseleko kunye nokusebenza kakuhle kwe-Rezdiffra kwavavanywa ngokusekelwe kuhlalutyo lwe-surrogate endpoint kwinyanga ye-12 kwinyanga ye-54, i-randomized, i-double blind blind-controlled controlled placebo. Isiphelo somlingane walinganisa ubungakanani be isibindi ukudumba kunye namanxeba. Umxhasi kufuneka aqhube isifundo emva kokuvunywa ukuqinisekisa nokuchaza inzuzo yekliniki ye-Rezdiffra, eya kwenziwa ngokugqiba isifundo esifanayo seenyanga ze-54, eqhubekayo. Ukubhalisa kulingo, izigulane kwakufuneka zibe ne- isibindi I-biopsy ebonisa ukudumba ngenxa ye-NASH ephakathi okanye ephezulu isibindi amanxeba. Kwilingo, izifundo ze-888 zabelwa ngokungaqhelekanga ukuba zifumane enye yezi zilandelayo: indawo ye-placebo (izifundo ze-294); I-80 milligrams yeRezdiffra (izifundo ezingama-298); okanye 100 milligrams of Rezdiffra (296 izifundo); kanye yonke imihla, ukongeza kukhathalelo oluqhelekileyo lwe-NASH, olubandakanya iingcebiso malunga nokutya okunempilo kunye nokuzilolonga.
Kwiinyanga ze-12, i-biopsies yesibindi ibonise ukuba inxalenye enkulu yezifundo eziye zanyangwa nge-Rezdiffra ziphumelele kwisisombululo se-NASH okanye ukuphuculwa kwesibindi xa kuthelekiswa nabo bafumana i-placebo. Itotali ye-26% ukuya kwi-27% yezifundo ezifumene i-80 milligrams ye-Rezdiffra kunye ne-24% ukuya kwi-36% yezifundo ezifumene i-100 milligrams ye-Rezdiffra bafumana isisombululo se-NASH kwaye akukho konakala kwesibindi, xa kuthelekiswa ne-9% ukuya kwi-13% yabo bathe bafumana i-23 milligrams ye-Rezdiffra. wafumana i-placebo kunye neengcebiso malunga nokutya kunye nokuzilolonga. Uluhlu lweempendulo lubonisa ufundo lwee-pathologists ezahlukeneyo. Ukongeza, i-80% yezifundo ezifumene i-24 milligrams ye-Rezdiffra kunye ne-28% ukuya kwi-100% yezifundo ezifumene i-XNUMX milligrams ye-Rezdiffra ziye zaphucuka. isibindi ukonakala kwaye kungabikho ukwanda kwe-NASH, xa kuthelekiswa ne-13% ukuya kwi-15% yabo bafumana i-placebo, kuxhomekeke kufundo lwe-pathologist nganye. Ukubonakaliswa kolu tshintsho kumlinganiselo wezigulane emva konyaka omnye wonyango kuyaphawuleka, njengoko isifo Iqhubela phambili kancinci kwaye uninzi lwezigulane luthatha iminyaka okanye amashumi eminyaka ukubonisa ukuqhubela phambili.
Iziphumo ebezingalindelekanga zeRezdiffra
Ezona ziphumo zixhaphakileyo zeRezdiffra ziquka urhudo kunye nesicaphucaphu. I-Rezdiffra iza nezilumkiso ezithile kunye nezilumkiso, ezinje ngetyhefu yesibindi esenziwe ngamachiza kunye neziphumo ebezingalindelekanga ezinxulumene ne-gallbladder.
Ukusetyenziswa kwe-Rezdiffra kufuneka kuphetshwe kwizigulane ezine-cirrhosis ethotyiweyo. Izigulana kufuneka ziyeke ukusebenzisa i-Rezdiffra ukuba ziphuhlisa iimpawu okanye iimpawu zokuba mandundu isibindi umsebenzi ngelixa kunyango lwe-Rezdiffra.
Ukusebenzisana kweziyobisi kweRezdiffra
Ukusebenzisa i-Rezdiffra ngaxeshanye njengamanye amachiza athile, ngakumbi amayeza okunciphisa i-cholesterol, kunokubangela intsebenziswano ebalulekileyo yamachiza. Ababoneleli bezempilo kufuneka babhekiselele kwingcaciso epheleleyo yokumisela ulwazi olongezelelweyo malunga nolu nxibelelwano lwamachiza olunokuba lubalulekileyo kunye ne-Rezdiffra, idosi ecetyiswayo kunye nokuguqulwa kolawulo.
The FDA i-Rezdiffra evunyiweyo phantsi kwendlela yokuvunywa okukhawulezileyo, evumela ukuvunywa kwangaphambili kwamachiza anyanga iimeko ezimandundu kunye nokujongana nesidingo sonyango esingafezekiswanga, ngokusekwe kwi-surrogate okanye i-intermediate endpoint yeklinikhi enokuthi iqikelele ngengqiqo inzuzo yeklinikhi. Uphononongo olufunekayo olukhankanywe ngasentla lweenyanga ze-54, oluqhubekayo, luya kuhlola inzuzo yekliniki emva kweenyanga ze-54 zonyango lwe-Rezdiffra.
I-Rezdiffra ifumene i-Breakthrough Therapy, i-Fast Track kunye ne-Priority Review ukutyunjwa kolu phawu.
The FDA inike imvume yeRezdiffra kwiMadrigal Pharmaceuticals.
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umthombo:
I-FDA 2024. Ukukhutshwa kweendaba - I-FDA ivuma unyango lokuQala lweZigulana ezineNyoko yesibindi ngenxa yeSifo sesibindi esinamafutha. Ithunyelwe nge-14 kaMatshi 2024. Iyafumaneka e https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-patients-liver-scarring-due-fatty-liver-disease
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