广告投放

Rezdiffra(resmetirom): FDA 批准首个治疗脂肪肝引起的肝脏疤痕的药物 

Rezdiffra(resmetirom)已被美国 FDA 批准用于治疗患有中度至晚期肝脏疤痕(纤维化)的成人非肝硬化非酒精性脂肪性肝炎(NASH),并与饮食和运动一起使用。  

到目前为止,对于肝脏有明显疤痕的非肝硬化非酒精性脂肪性肝炎 (NASH) 患者,还没有一种药物可以直接解决他们的问题。 肝损害. FDA的 Rezdiffra 的批准将首次为这些患者提供除饮食和运动之外的治疗选择。  

NASH is a result of the progression of non-alcoholic fatty disease where 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 是甲状腺激素受体的部分激活剂; Rezdiffra 在肝脏中激活该受体可减少肝脏脂肪积累。  

Rezdiffra 的安全性和功效  

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 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 biopsy showing inflammation due to NASH with moderate or advanced 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 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 disease typically progresses slowly with most patients taking years or even decades to show progression. 

Rezdiffra 的副作用  

Rezdiffra 最常见的副作用包括腹泻和恶心。 Rezdiffra 带有某些警告和预防措施,例如药物引起的肝毒性和胆囊相关的副作用。  

Use of Rezdiffra should be avoided in patients with decompensated cirrhosis. Patients should stop using Rezdiffra if they develop signs or symptoms of worsening function while on Rezdiffra treatment.  

Rezdiffra的药物相互作用  

将 Rezdiffra 与某些其他药物(特别是用于降低胆固醇的他汀类药物)同时使用可能会导致潜在的显着药物相互作用。医疗保健提供者应参阅完整的处方信息,以获取有关 Rezdiffra 与这些潜在重要药物相互作用、推荐剂量和给药修改的更多信息。  

FDA Rezdiffra 在加速批准途径下获得批准,该途径允许根据合理可能预测临床获益的替代或中间临床终点,提前批准治疗严重疾病和解决未满足的医疗需求的药物。上述所需的 54 个月研究正在进行中,将评估 Rezdiffra 治疗 54 个月后的临床益处。  

Rezdiffra 获得了针对该适应症的突破性疗法、快速通道和优先审查资格。  

FDA 向 Madrigal Pharmaceuticals 授予 Rezdiffra 的批准。 

*** 

Sumber: 

FDA 2024。新闻稿 – FDA 批准首次治疗脂肪肝引起的肝脏疤痕患者。发布于 14 年 2024 月 XNUMX 日。可在 https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-patients-liver-scarring-due-fatty-liver-disease 

*** 

赛欧团队
赛欧团队https://www.ScientificEuropean.co.uk
科学欧洲® | SCIEU.com | 科学的重大进步。 对人类的影响。 鼓舞人心。

订阅电邮通讯

将通过所有最新新闻,优惠和特别公告进行更新。

最热门文章

印度的 COVID-19 危机:可能出了什么问题

印度当前危机的成因分析...

鼻凝胶:一种含有 COVID-19 的新方法

使用鼻凝胶作为一种新方法来...

BrainNet:第一个直接“脑对脑”交流的案例

科学家们首次展示了多人...
- 广告 -
94,514风扇喜欢
47,678产品粉丝关注
1,772产品粉丝关注
30认购订阅