USA:n FDA on hyväksynyt Rezdiffran (resmetiromin) aikuisten ei-kirroottisen alkoholittoman steatohepatiitin (NASH) hoitoon, jossa on kohtalainen tai pitkälle edennyt maksan arpeutuminen (fibroosi), käytettäväksi ruokavalion ja liikunnan kanssa.
Tähän asti ei-kirroottista alkoholitonta steatohepatiittia (NASH) sairastavilla potilailla, joilla on myös huomattavaa maksan arpeutumista, ei ole ollut lääkitystä, joka voisi suoraan hoitaa heidän maksavaurio. FDA: n approval of Rezdiffra will, for the first time, provide a hoito option for these patients, in addition to diet and exercise.
NASH is a result of the progression of non-alcoholic fatty maksa tauti jossa maksa 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 kilpirauhashormonireseptorin osittainen aktivaattori; Rezdiffra aktivoi tämän reseptorin maksassa, mikä vähentää maksan rasvan kertymistä.
Rezdiffran turvallisuus ja teho
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 maksa 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 maksa biopsy showing inflammation due to NASH with moderate or advanced maksa 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 maksa 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 tauti typically progresses slowly with most patients taking years or even decades to show progression.
Rezdiffran sivuvaikutukset
Rezdiffran yleisimmät sivuvaikutukset olivat ripuli ja pahoinvointi. Rezdiffra sisältää tiettyjä varoituksia ja varotoimia, kuten lääkkeiden aiheuttamaa maksatoksisuutta ja sappirakkoon liittyviä sivuvaikutuksia.
Use of Rezdiffra should be avoided in patients with decompensated cirrhosis. Patients should stop using Rezdiffra if they develop signs or symptoms of worsening maksa function while on Rezdiffra treatment.
Rezdiffran lääkevuorovaikutukset
Rezdiffran käyttö samanaikaisesti tiettyjen muiden lääkkeiden, erityisesti kolesterolia alentavien statiinien, kanssa voi johtaa mahdollisesti merkittäviin lääkeinteraktioihin. Terveydenhuollon tarjoajien tulee katsoa täydellisiä reseptitietoja saadakseen lisätietoja näistä mahdollisesti merkittävistä lääkkeiden yhteisvaikutuksista Rezdiffran kanssa, suositelluista annostuksista ja annostelun muutoksista.
- 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 Breakthrough Therapy-, Fast Track- ja Priority Review -nimitykset tästä käyttöaiheesta.
- FDA granted the approval of Rezdiffra to Madrigal Pharmaceuticals.
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Lähde:
FDA 2024. Lehdistötiedote – FDA hyväksyy ensimmäisen hoidon potilaille, joilla on rasva-maksasairauden aiheuttama maksan arpeutuminen. Julkaistu 14. maaliskuuta 2024. Saatavilla osoitteessa https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-patients-liver-scarring-due-fatty-liver-disease
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