Rezdiffra (resmetirom) hefur verið samþykkt af FDA í Bandaríkjunum til meðferðar á fullorðnum með óáfenga fituhrörnunarbólgu (NASH) með miðlungs til langt gengið lifrarör (fibrosis), til notkunar ásamt mataræði og hreyfingu.
Hingað til hafa sjúklingar með ekki skorpulifur óáfenga lifrarbólgu (NASH) sem einnig eru með áberandi lifrarör, ekki haft lyf sem gæti beint lifrarskemmdir. FDA approval of Rezdiffra will, for the first time, provide a meðferð option for these patients, in addition to diet and exercise.
NASH is a result of the progression of non-alcoholic fatty lifur Sjúkdómurinn þar sem lifur inflammation, over time, can lead to liver scarring and liver dysfunction. NASH is often associated with other health problems such as high blood pressure and type 2 diabetes. By at least one estimate, approximately 6-8 million people in the U.S. have NASH with moderate to advanced liver scarring, with that number expected to increase.
Rezdiffra er að hluta virkjaður skjaldkirtilshormónsviðtaka; virkjun þessa viðtaka af Rezdiffra í lifur dregur úr uppsöfnun lifrarfitu.
Öryggi og verkun Rezdiffra
The safety and efficacy of Rezdiffra was evaluated based on an analysis of a surrogate endpoint at month 12 in a 54-month, randomized, double-blind placebo-controlled trial. The surrogate endpoint measured the extent of lifur inflammation and scarring. The sponsor is required to conduct a postapproval study to verify and describe Rezdiffra’s clinical benefit, which will be done through completing the same 54-month study, which is still ongoing. To enroll in the trial, patients needed to have a lifur biopsy showing inflammation due to NASH with moderate or advanced lifur scarring. In the trial, 888 subjects were randomly assigned to receive one of the following: placebo (294 subjects); 80 milligrams of Rezdiffra (298 subjects); or 100 milligrams of Rezdiffra (296 subjects); once daily, in addition to standard care for NASH, which includes counseling for healthy diet and exercise.
At 12 months, liver biopsies showed that a greater proportion of subjects who were treated with Rezdiffra achieved NASH resolution or an improvement in liver scarring as compared with those who received the placebo. A total of 26% to 27% of subjects who received 80 milligrams of Rezdiffra and 24% to 36% of subjects who received 100 milligrams of Rezdiffra experienced NASH resolution and no worsening of liver scarring, compared to 9% to 13% of those who received placebo and counseling on diet and exercise. The range of responses reflects different pathologists’ readings. In addition, a total of 23% of subjects who received 80 milligrams of Rezdiffra and 24% to 28% of subjects who received 100 milligrams of Rezdiffra experienced an improvement in lifur scarring and no worsening of NASH, compared to 13% to 15% of those who received placebo, depending on each pathologist’s readings. Demonstration of these changes in a proportion of patients after just one year of treatment is notable, as the Sjúkdómurinn typically progresses slowly with most patients taking years or even decades to show progression.
Aukaverkanir af Rezdiffra
Algengustu aukaverkanir Rezdiffra voru niðurgangur og ógleði. Rezdiffra kemur með ákveðnar viðvaranir og varúðarráðstafanir, svo sem eiturverkanir á lifur af völdum lyfja og gallblöðru-tengdar aukaverkanir.
Use of Rezdiffra should be avoided in patients with decompensated cirrhosis. Patients should stop using Rezdiffra if they develop signs or symptoms of worsening lifur function while on Rezdiffra treatment.
Lyfjamilliverkanir Rezdiffra
Notkun Rezdiffra á sama tíma og ákveðin önnur lyf, einkum statín til að lækka kólesteról, getur leitt til hugsanlegra marktækra lyfjamilliverkana. Heilbrigðisstarfsmenn ættu að vísa til allra ávísanaupplýsinga til að fá frekari upplýsingar um þessar hugsanlegu mikilvægu lyfjamilliverkanir við Rezdiffra, ráðlagða skammta og breytingar á lyfjagjöf.
The FDA approved Rezdiffra under the accelerated approval pathway, which allows for earlier approval of drugs that treat serious conditions and address an unmet medical need, based on a surrogate or intermediate clinical endpoint that is reasonably likely to predict clinical benefit. The required aforementioned 54-month study, which is ongoing, will assess clinical benefit after 54 months of Rezdiffra treatment.
Rezdiffra fékk tilnefningarnar Breakthrough Therapy, Fast Track og Priority Review fyrir þessa ábendingu.
The FDA granted the approval of Rezdiffra to Madrigal Pharmaceuticals.
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Heimild:
FDA 2024. Fréttatilkynning – FDA samþykkir fyrstu meðferð fyrir sjúklinga með lifrarör vegna fitusjúkdóms í lifur. Sent 14. mars 2024. Fæst á https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-patients-liver-scarring-due-fatty-liver-disease
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