Intercept Pharmaceuticals, Inc., a biopharmaceutical company and wholly owned subsidiary of Alfasigma S.p.A. focused on the development and commercialization of novel therapeutics to treat rare and serious liver diseases, today announced that the U.S.

生物制药公司Intercept Pharmaceuticals，Inc.，Alfasigma S.p.a.的全资子公司，专注于治疗罕见和严重肝病的新型疗法的开发和商业化，今天宣布美国。

Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for Ocaliva for the treatment of individuals with primary biliary cholangitis (PBC). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of October 15, 2024. In this communication, the agency informed Intercept that they are planning to hold an Advisory Committee meeting to discuss the application.

美国食品和药物管理局（FDA）已接受其奥卡利娃补充新药申请（sNDA），用于治疗原发性胆汁性胆管炎（PBC）患者。美国食品和药物管理局（FDA）已将处方药使用费法案（PDUFA）的目标行动日期指定为2024年10月15日。在这封信中，该机构告知Intercept，他们计划举行一次咨询委员会会议，讨论该申请。

“The sNDA data submission asserted that, with more than seven years on the market and more than 40,000 patient-years of post-marketing experience in PBC, Ocaliva is the only second-line therapy that has multiple published analyses of registry data and real-world evidence showing significant effect on transplant-free and decompensation-free survival in people living with PBC,” said Paul Nitschmann, M.D., Senior Vice President of Regulatory Affairs at Intercept.

“sNDA提交的数据声称，Ocaliva在PBC上市已有七年多的时间，在上市后有40000多年的经验，是唯一一种二线治疗方法，它对登记数据和现实世界的证据进行了多次分析，显示出对PBC患者的无移植和无失代偿生存率有显着影响，”Intercept监管事务高级副总裁保罗·尼施曼（PaulNitschmann）医学博士表示。

“We are pleased that the FDA has filed the submission and look forward to continued, constructive interactions with the agency, including discussing our precedent-setting application during the forthcoming Advisory Committee meeting.”

“我们很高兴FDA提交了申请，并期待着与该机构继续进行建设性的互动，包括在即将举行的咨询委员会会议上讨论我们的先例设置申请。”

Ocaliva received accelerated approval in 2016 and is indicated for the treatment of adult patients living with primary biliary cholangitis (PBC) without cirrhosis or with compensated cirrhosis who do not have evidence of portal hypertension, either in combination with ursodeoxycholic acid (UDCA) with an inadequate response to UDCA or as monotherapy in patients unable to tolerate UDCA.

Ocaliva于2016年获得加速批准，用于治疗无肝硬化或代偿性肝硬化的原发性胆汁性胆管炎（PBC）成年患者，这些患者没有门静脉高压的证据，或者与熊去氧胆酸（UDCA）联合使用，对UDCA的反应不足，或者作为无法耐受UDCA的患者的单一疗法。

The precedent-setting sNDA for Ocaliva is intended to satisfy the post-marketing requirements to confirm a clinical benefit in patients with PBC. The sNDA is supported by data from the Company’s post-marketing requirement studies COBALT and Study 401 as well as real-world evidence from a U.S. claims database and international PBC patient registries.

Ocaliva的先例sNDA旨在满足上市后的要求，以确认PBC患者的临床益处。sNDA得到了该公司上市后需求研究COBALT和研究401的数据以及美国索赔数据库和国际PBC患者登记处的真实证据的支持。

Intercept and Alfasigma remain committed to supporting people living with PBC and look forward to advancing discussions with the agency.

Intercept和Alfasigma仍致力于支持与PBC生活在一起的人，并期待与该机构进行讨论。

About Primary Biliary Cholangitis

关于原发性胆汁性胆管炎

Primary biliary cholangitis (PBC) is a rare, progressive, and chronic autoimmune disease that affects the bile ducts in the liver and is most prevalent (approximately 1 in 10,000) in women over the age of 40. PBC causes bile acid to build up in the liver, resulting in inflammation and scarring (fibrosis), which, if left untreated, can lead to cirrhosis, a liver transplant, or death.

原发性胆汁性胆管炎（PBC）是一种罕见的，进行性的慢性自身免疫性疾病，会影响肝脏中的胆管，在40岁以上的女性中最为普遍（约10000分之一）。PBC导致胆汁酸在肝脏中积聚，导致炎症和瘢痕形成（纤维化），如果不及时治疗，可能导致肝硬化，肝移植或死亡。

About Ocaliva® (obeticholic acid)

关于Ocaliva®（奥贝胆酸）

OCALIVA, a farnesoid X receptor (FXR) agonist, is indicated for the treatment of adult patients with primary biliary cholangitis (PBC)

OCALIVA是一种法尼醇X受体（FXR）激动剂，适用于治疗成人原发性胆汁性胆管炎（PBC）

without cirrhosis or

无肝硬化或

with compensated cirrhosis who do not have evidence of portal hypertension, either in combination with ursodeoxycholic acid (UDCA) with an inadequate response to UDCA or as monotherapy in patients unable to tolerate UDCA.

代偿性肝硬化患者没有门静脉高压的证据，或者与熊去氧胆酸（UDCA）联合使用，对UDCA的反应不足，或者作为不能耐受UDCA的患者的单一疗法。

This indication is approved under accelerated approval based on a reduction in alkaline phosphatase (ALP). An improvement in survival or disease-related symptoms has not been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

基于碱性磷酸酶（ALP）的减少，该适应症在加速批准下获得批准。生存或疾病相关症状的改善尚未确定。是否继续批准该适应症可能取决于验证性试验中临床益处的验证和描述。

About Intercept

关于Intercept

Intercept is a biopharmaceutical company and a wholly owned subsidiary of Alfasigma S.p.A. focused on the development and commercialization of novel therapeutics to treat rare and serious liver diseases, including primary biliary cholangitis (PBC) and severe alcohol-associated hepatitis (sAH). Intercept owns the commercial rights to Ocaliva in the U.S.market.

Intercept是一家生物制药公司，是Alfasigma S.p.a.的全资子公司，专注于开发和商业化治疗罕见和严重肝病的新型疗法，包括原发性胆汁性胆管炎（PBC）和严重酒精相关性肝炎（sAH）。Intercept拥有Ocaliva在美国市场的商业权利。