FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has approved LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients five years of age and older with progressive familial intrahepatic cholestasis (PFIC).

加利福尼亚州福斯特城（商业新闻短讯）--Mirum Pharmaceuticals，Inc.（纳斯达克股票代码：MIRM）今天宣布，美国食品和药物管理局（FDA）已批准LIVMARLI®（maralixibat）口服溶液用于治疗5岁及以上进行性家族性肝内胆汁淤积症（PFIC）患者的胆汁淤积性瘙痒症。

Mirum has also submitted an additional supplemental new drug application (sNDA) to introduce a higher concentration formulation of LIVMARLI, used during the MARCH study, to enable label expansion for younger patients with PFIC, later this year..

Mirum还提交了一份额外的补充新药申请（sNDA），以引入三月研究期间使用的更高浓度的利夫马利制剂，以便在今年晚些时候为年轻的PFIC患者扩大标签。

LIVMARLI is also approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. (three months and older), Europe (two months and older), in Canada and other regions globally.

LIVMARLI还被批准用于治疗美国（三个月及以上），欧洲（两个月及以上），加拿大和全球其他地区的Alagille综合征（ALGS）患者的胆汁淤积性瘙痒症。

“LIVMARLI has the potential to have a transformational impact for patients with cholestatic pruritus associated with PFIC, and importantly, offers an option for those patients with the rarest of subtypes,” said Chris Peetz, chief executive officer at Mirum. “Thank you to the patients, families, and clinicians whose study participation made this approval possible.”.

Mirum首席执行官克里斯·佩茨（ChrisPeetz）表示：“利夫马利（LIVMARLI）有可能对PFIC相关的胆汁淤积性瘙痒症患者产生变革性影响，重要的是，它为那些亚型最罕见的患者提供了一种选择。”。“感谢患者，家属和临床医生，他们的研究参与使这一批准成为可能。”

The approval is based on data from the Phase 3 MARCH study, the largest randomized trial conducted in PFIC, with 93 patients across a range of genetic PFIC types, including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6, and unidentified mutational status.

该批准基于3月3期研究的数据，该研究是在PFIC中进行的最大规模的随机试验，共有93名患者参与了一系列遗传PFIC类型，包括PFIC1，PFIC2，PFIC3，PFIC4，PFIC6和未知突变状态。

“LIVMARLI’s approval in cholestatic pruritus for patients with PFIC is a result of years of investigation and a collection of a strong body of clinical evidence showing meaningful improvements across a number of important parameters, including pruritus, affecting children with PFIC,” said Richard Thompson, professor of molecular hepatology at King’s College London and an investigator in the MARCH study.

理查德·汤普森（Richard Thompson）说：“利夫马利（LIVMARLI）批准PFIC患者使用胆汁淤积性瘙痒症是多年调查的结果，大量临床证据表明，包括瘙痒症在内的许多重要参数都有显着改善，影响了PFIC儿童。”，伦敦国王学院分子肝病学教授和三月研究的研究员。

“I am pleased that we will have a well-studied and efficacious option to offer patients whose life has been disrupted by itch.”.

“我很高兴我们将有一个经过充分研究和有效的选择，为那些生活被瘙痒打断的患者提供服务。”

“PFIC is a difficult disease for both the patient and family and significantly impedes the quality of life for all,” said Emily Ventura, executive director of the PFIC Network and mom to a daughter with PFIC. “These young and fragile patients endure an itch so severe that they experience deficits in their sleep, nutrition, growth, and, in the past, some have turned to transplant to resolve their itch.

“PFIC对患者和家人来说都是一种困难的疾病，严重影响了所有人的生活质量，”PFIC网络执行董事艾米丽·文图拉（EmilyVentura）说，她是PFIC女儿的母亲。“这些年轻而脆弱的患者忍受着非常严重的瘙痒，以至于他们在睡眠、营养、生长方面都出现了缺陷，并且在过去，一些人已经转向移植来解决他们的瘙痒。

We are hopeful that LIVMARLI will have a measurable impact for patients, potentially offering a new normal and relief from the sleepless nights and disruption to their lives caused by cholestatic pruritus.”.

我们希望LIVMARLI将对患者产生可测量的影响，有可能为患者提供一种新的正常状态，缓解因胆汁淤积性瘙痒症引起的失眠和生活中断。”

About PFIC

关于PFIC

Progressive familial intrahepatic cholestasis (PFIC) is a rare genetic disorder that causes progressive liver disease typically leading to liver failure. In people with PFIC, liver cells are less able to secrete bile. The resulting buildup of bile causes liver disease in affected individuals. Signs and symptoms of PFIC typically begin in infancy.

进行性家族性肝内胆汁淤积症（PFIC）是一种罕见的遗传疾病，可导致进行性肝病，通常会导致肝衰竭。在PFIC患者中，肝细胞分泌胆汁的能力较差。由此产生的胆汁积聚会导致受影响个体的肝病。PFIC的体征和症状通常始于婴儿期。

Patients experience severe itching, jaundice, failure to grow at the expected rate (failure to thrive), and an increasing inability of the liver to function (liver failure). The disease is estimated to affect one in every 50,000 to 100,000 births in the United States and Europe. More than six types of PFIC have been genetically identified, all of which are similarly characterized by impaired bile flow and progressive liver disease..

患者会出现严重的瘙痒，黄疸，无法以预期的速度生长（无法failure壮成长），以及肝脏越来越无法发挥功能（肝衰竭）。据估计，在美国和欧洲，每5万至10万名新生儿中就有一名患有这种疾病。已经通过基因鉴定出六种以上的PFIC，所有这些PFIC的特征都是胆汁流量受损和进行性肝病。

About LIVMARLI® (maralixibat) oral solution

关于LIVMARLI®（马利昔巴特）口服液

LIVMARLI® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor and the only approved medication by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) three months of age and older and progressive familial intrahepatic cholestasis (PFIC) five years of age and older..

LIVMARLI®（maralixibat）口服溶液是一种口服给药的回肠胆汁酸转运蛋白（IBAT）抑制剂，每日一次，是美国食品和药物管理局唯一批准用于治疗三个月及以上Alagille综合征（ALGS）和五岁及以上进行性家族性肝内胆汁淤积症（PFIC）患者胆汁淤积性瘙痒症的药物。

LIVMARLI is also the only approved IBAT inhibitor approved by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months and older, and by Health Canada for the treatment of cholestatic pruritus in ALGS. For more information for U.S. residents, please visit LIVMARLI.com..

LIVMARLI也是唯一被欧盟委员会批准用于治疗两个月及以上ALGS患者胆汁淤积性瘙痒症的IBAT抑制剂，也是加拿大卫生部批准用于治疗ALGS胆汁淤积性瘙痒症的IBAT抑制剂。有关美国居民的更多信息，请访问LIVMARLI.com。

Mirum has also submitted LIVMARLI for approval in Europe in PFIC for patients two months of age and older.

Mirum还向欧洲PFIC提交了两个月及以上患者的LIVMARLI申请批准。

LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS and PFIC. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website.

LIVMARLI已获得ALGS和PFIC 2型的突破性治疗指定以及ALGS和PFIC的孤儿指定。要了解有关LIVMARLI正在进行的临床试验的更多信息，请访问该公司网站上的Mirum临床试验部分。

About Mirum Pharmaceuticals, Inc.

关于Mirum Pharmaceuticals, Inc.

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 five years of age and older. Mirum has submitted for approval in Europe for the treatment of PFIC in patients two months of age and older. 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.

它在美国也被批准用于五岁及以上PFIC患者的胆汁淤积性瘙痒症。Mirum已提交欧洲批准，用于治疗两个月及以上患者的PFIC。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 has received medical necessity recognition by the FDA to treat patients with cerebrotendinous xanthomatosis (CTX)..

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 2b VISTAS study for primary sclerosing cholangitis 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的2b期VISTAS研究和原发性胆汁性胆管炎的2b期VANTAGE研究。

Lastly, CHENODAL, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023..

最后，CHENODAL已在一项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。