FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to volixibat as a potential treatment for cholestatic pruritus in patients with primary biliary cholangitis (PBC).

加利福尼亚州福斯特城（商业新闻短讯）--Mirum Pharmaceuticals，Inc.（纳斯达克：MIRM）今天宣布，美国食品和药物管理局（FDA）已授予volixibat突破性治疗指定，作为原发性胆汁性胆管炎（PBC）患者胆汁淤积性瘙痒症的潜在治疗方法。

The regulatory designation is based on the positive interim analysis of the Phase 2b VANTAGE study which showed statistically significant improvement versus placebo in pruritus for patients treated with volixibat. The confirmatory portion of the study is ongoing with completion of enrollment expected in 2026..

监管指定是基于2b期VANTAGE研究的积极中期分析，该研究显示，与安慰剂相比，接受volixibat治疗的患者瘙痒症有统计学显着改善。该研究的确认部分正在进行中，预计将于2026年完成入学。。

“Breakthrough Therapy Designation for volixibat in PBC underscores the importance and urgency for a treatment to address one of the most burdensome impacts of this rare liver disease,” said Joanne Quan, MD, chief medical officer at Mirum. “We look forward to advancing our VANTAGE study with the goal of making volixibat available to patients living with PBC-related itch as quickly as possible.”.

Mirum首席医疗官Joanne Quan医学博士说：“PBC中volixibat的突破性治疗指定强调了治疗的重要性和紧迫性，以解决这种罕见肝病最沉重的影响之一。”。“我们期待着推进我们的VANTAGE研究，目标是尽快为患有PBC相关瘙痒的患者提供volixibat。”。

The FDA’s Breakthrough Therapy Designation aims to expedite the development and review of drugs that are intended to treat serious or life-threatening diseases. To qualify for this designation, preliminary clinical evidence must indicate that the drug may demonstrate substantial improvement over existing therapy on at least one clinically significant endpoint..

FDA的突破性治疗指定旨在加快旨在治疗严重或危及生命的疾病的药物的开发和审查。为了有资格获得这一称号，初步临床证据必须表明，该药物可能在至少一个具有临床意义的终点上比现有疗法有实质性改善。。

About Volixibat

关于Volixibat

Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver.

Volixibat是一种口服，最小吸收的药物，旨在选择性抑制回肠胆汁酸转运蛋白（IBAT）。Volixibat可能通过抑制IBAT阻断胆汁酸的循环，从而全身和肝脏中的胆汁酸减少，从而为治疗成人胆汁淤积性疾病提供了一种新方法。

Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (VISTAS study), and primary biliary cholangitis (VANTAGE study). In June, Mirum announced positive interim results from the Phase 2b VANTAGE study showing statistically significant improvement in pruritus as well as meaningful reductions in serum bile acids and improvements in fatigue for patients treated with volixibat.

Volixibat目前正在原发性硬化性胆管炎（VISTAS研究）和原发性胆汁性胆管炎（VANTAGE研究）的2b期研究中进行评估。6月，Mirum宣布了2b期VANTAGE研究的积极中期结果，显示瘙痒有统计学意义的改善，血清胆汁酸有意义的降低，接受volixibat治疗的患者的疲劳有所改善。

No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate..

没有观察到新的安全信号，最常见的不良事件是腹泻，所有病例均为轻度至中度。。

About Mirum Pharmaceuticals, Inc.

关于Mirum Pharmaceuticals，股份有限公司。

Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution, CHOLBAM® (cholic acid) capsules, and CHENODAL® (chenodiol) tablets.

Mirum Pharmaceuticals，Inc.是一家生物制药公司，致力于改变影响儿童和成人的罕见疾病的治疗方法。Mirum有三种批准的药物：LIVMARLI®（maralixibat）口服溶液，CHOLBAM®（胆酸）胶囊和CHENODAL®（chenodiol）片剂。

LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally.

LIVMARLI是一种IBAT抑制剂，被批准用于治疗影响儿童和成人的两种罕见肝病。在美国（三个月及以上），欧洲（两个月及以上）和全球其他地区，它被批准用于治疗Alagille综合征患者的胆汁淤积性瘙痒症。

It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum is also initiating the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus.

它在美国也被批准用于12个月及以上的PFIC患者的胆汁淤积性瘙痒症；。Mirum还启动了3期EXPAND研究，这是LIVMARLI在胆汁淤积性瘙痒症的其他情况下的标签扩展机会。

CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms or liver disease. CHENODAL has received medical necessity recognition by the FDA to treat patients with cerebrotendinous xanthomatosis (CTX)..

CHOLBAM被FDA批准用于治疗由于单酶缺乏引起的胆汁酸合成障碍，以及对表现出体征或症状或肝病的患者进行过氧化物酶体疾病的辅助治疗。CHENODAL已获得FDA的医疗必要性认可，用于治疗脑腱黄瘤病（CTX）患者。。

Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis.

Mirum的晚期管道包括两种用于治疗衰弱性肝病的研究性治疗方法。IBAT抑制剂Volixibat正在两项潜在的注册研究中进行评估，包括原发性硬化性胆管炎（PSC）的2期VISTAS研究和原发性胆汁性胆管炎的2b期VANTAGE研究。

Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Lastly, chenodiol, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023. Mirum has submitted a new drug application with the FDA for the approval of chenodiol to treat CTX in the U.S..

Volixibat已被授予突破性治疗指定，用于治疗PBC患者的胆汁淤积性瘙痒症。最后，chenodiol已在3期临床研究RESTORE中进行了评估，以治疗CTX患者，2023年报告的结果为阳性。