Rezdiffra (resmetirom) on USA FDA poolt heaks kiidetud mittetsirroosilise mittealkohoolse steatohepatiidi (NASH) täiskasvanute raviks koos mõõduka kuni kaugelearenenud maksaarmistumisega (fibroos), mida kasutatakse koos dieedi ja treeninguga.
Seni ei olnud mittetsirrootilise mittealkohoolse steatohepatiidi (NASH) patsientidel, kellel on ka märkimisväärne maksaarmistumine, ravimeid, mis võiksid nende haigust otseselt ravida. maksakahjustuse. FDA-d approval of Rezdiffra will, for the first time, provide a ravi option for these patients, in addition to diet and exercise.
NASH is a result of the progression of non-alcoholic fatty maks haigus kus maks 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 on kilpnäärme hormooni retseptori osaline aktivaator; selle retseptori aktiveerimine Rezdiffra poolt maksas vähendab rasva kogunemist maksas.
Rezdiffra ohutus ja efektiivsus
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 maks 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 maks biopsy showing inflammation due to NASH with moderate or advanced maks 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 maks 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 haigus typically progresses slowly with most patients taking years or even decades to show progression.
Rezdiffra kõrvaltoimed
Rezdiffra kõige sagedasemad kõrvalnähud olid kõhulahtisus ja iiveldus. Rezdiffra sisaldab teatud hoiatusi ja ettevaatusabinõusid, nagu ravimitest põhjustatud maksatoksilisus ja sapipõiega seotud kõrvaltoimed.
Use of Rezdiffra should be avoided in patients with decompensated cirrhosis. Patients should stop using Rezdiffra if they develop signs or symptoms of worsening maks function while on Rezdiffra treatment.
Rezdiffra ravimite koostoimed
Rezdiffra kasutamine samaaegselt teatud teiste ravimitega, eriti kolesterooli alandavate statiinidega, võib põhjustada potentsiaalselt olulisi ravimite koostoimeid. Tervishoiuteenuse osutajad peaksid lugema täielikku väljakirjutamisteavet, et saada lisateavet nende potentsiaalselt oluliste ravimite koostoimete kohta Rezdiffraga, soovitatava annuse ja manustamise muudatuste kohta.
. 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 sai selle näidustuse kohta läbimurderavi, kiirravi ja prioriteetse läbivaatamise nimetused.
. FDA granted the approval of Rezdiffra to Madrigal Pharmaceuticals.
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Allikas:
FDA 2024. Pressiteade – FDA kiitis heaks rasvmaksahaigusest tingitud maksaarmistumisega patsientide esimese ravi. Postitatud 14. märtsil 2024. Saadaval aadressil https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-patients-liver-scarring-due-fatty-liver-disease
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