FDA批准Crinetics制药公司首款用于罕见生长障碍的每日口服药-动脉网

FDA Approves Crinetics Pharma's First Daily Pill For Rare Growth Disorder

benzinga 等信源发布 2025-09-26 13:07

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On Thursday, the U.S. Food and Drug Administration (FDA) approved

周四，美国食品和药物管理局 (FDA) 批准了

Crinetics Pharmaceuticals, Inc.’s

克里内蒂克斯制药公司

(NASDAQ:

（纳斯达克：

CRNX

) Palsonify (paltusotine) for the first-line treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom

) Palsonify（帕图索汀）用于治疗对手术反应不足和/或不适合手术的成年肢端肥大症患者的一线治疗。

surgery is not an option

手术不是一种选择。

。

Acromegaly is a rare disorder characterized by the excessive production of growth hormone (GH), leading to abnormal growth of hands, feet, and facial features in adults.

肢端肥大症是一种罕见的疾病，其特征是生长激素（GH）的过度分泌，导致成年人的手、脚和面部特征异常增长。

Palsonify, a selectively-targeted somatostatin receptor type 2 nonpeptide (SST2) agonist, is now the first once-daily, oral treatment approved for adults with acromegaly.

帕尔索尼非（Palsonify），一种选择性靶向的生长抑素受体2型非肽类（SST2）激动剂，现已成为首个获批用于治疗成年肢端肥大症患者的每日一次口服药物。

Also Read:

另请阅读：

Crinetics Reports Durable Symptom, Hormone Control With Oral Drug In Acromegaly

Crinetics报告称口服药物在肢端肥大症中表现出持久的症状和激素控制效果

The approval is based on data from the PATHFNDR-1 and PATHFNDR-2 Phase 3 pivotal trials, which evaluated Palsonify’s safety and efficacy in previously treated and medically untreated adults with acromegaly. Across both trials, Palsonify consistently demonstrated rapid onset, reliable biochemical control, .

该批准基于 PATHFNDR-1 和 PATHFNDR-2 三期关键试验的数据，这些试验评估了 Palsonify 在既往接受过治疗和未经药物治疗的成年肢端肥大症患者中的安全性和有效性。在两项试验中，Palsonify 始终表现出快速起效、可靠的生化控制。

and sustained efficacy

持续的有效性

。

Participants also reported significant reductions in signs and symptoms associated with acromegaly as measured by the Acromegaly Symptom Diary (ASD), an FDA-aligned patient-reported outcome tool developed to capture the symptoms, including headaches, joint pain, sweating, fatigue, weakness, swelling, and/or numbness/tingling..

参与者还报告说，根据《肢端肥大症症状日记》（ASD）测量，与肢端肥大症相关的体征和症状显著减少。ASD是一种与FDA一致的患者报告结果工具，旨在捕捉包括头痛、关节疼痛、出汗、疲劳、虚弱、肿胀和/或麻木/刺痛等症状。

Palsonify was generally well-tolerated, with no serious adverse events reported in the randomized controlled portion of the trials.

Palsonify 通常具有良好的耐受性，在随机对照试验部分未报告严重不良事件。

Palsonify is expected to be available in the U.S. in early October.

Palsonify 预计将在十月初在美国上市。

A Marketing Authorization Application (MAA) for paltusotine in acromegaly is currently under review for use in the European Union, and the current timeline for the Committee for Medicinal Products and Human Use (CHMP) opinion is the first half of 2026.

目前，帕图索汀治疗肢端肥大症的上市许可申请（MAA）正在接受欧盟使用的审查，人用药品委员会（CHMP）意见的当前时间表为2026年上半年。

Crinetics is in partnership with Sanwa Kagaku Kenkyuso (SKK) to develop and commercialize paltusotine for acromegaly in Japan.

Crinetics与三和化学研究所（SKK）合作，在日本开发和商业化用于治疗肢端肥大症的paltusotine。

Paltusotine is also being evaluated for the treatment of carcinoid syndrome in the pivotal Phase 3 CAREFNDR trial.

Paltusotine 还在关键的 3 期 CAREFNDR 试验中被评估用于治疗类癌综合征。

Carcinoid syndrome is a collection of symptoms, including flushing, diarrhea, and breathing problems, caused by hormones and other chemicals released from a carcinoid tumor.

类癌综合征是一组症状，包括潮红、腹泻和呼吸问题，由类癌肿瘤释放的激素和其他化学物质引起。

Price Action:

价格行为:

CRNX stock was trading higher by 27.35% to $45.71 at last check Friday.

CRNX股票在上周五最后一次交易中上涨了27.35%，达到45.71美元。

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