BridgeBio Pharma has received

BridgeBio Pharma 已收到

European approval

欧洲批准

for a drug that treats cardiomyopathy stemming from a rare metabolic condition, strengthening the biotech’s position to take market share from the blockbuster Pfizer drug that is currently the standard treatment.

一种治疗由罕见代谢疾病引起的心肌病的药物，巩固了这家生物技术公司从目前作为标准治疗方案的辉瑞畅销药手中夺取市场份额的地位。

The European Commission granted marketing authorization to acoramidis, which BridgeBio developed as treatment for transthyretin amyloidosis (ATTR), a disease in which a genetic mutation leads to abnormal versions of the liver protein transthyretin (TTR). The misfolded proteins can cause nerve and heart problems.

欧盟委员会授予了acoramidis的上市许可，这是BridgeBio开发的一种用于治疗转甲状腺素蛋白淀粉样变性（ATTR）的药物，该疾病中的一种基因突变会导致肝脏蛋白质转甲状腺素蛋白（TTR）的异常版本。错误折叠的蛋白质可能会引起神经和心脏问题。

BridgeBio’s acoramidis, which will be commercialized in Europe as Beyonttra, is a small molecule designed to stabilize TTR to treat the cardiomyopathy caused by the disease. The biotech contends its drug is a better stabilizer than the drugs marketed by Pfizer. For 2024, Pfizer reported $3.3 billion in revenue from its Vyndaqel family of TTR stabilizers, a 36% increase compared to 2023..

BridgeBio的acoramidis，将在欧洲以Beyonttra的品牌进行商业化，它是一种旨在稳定TTR以治疗该疾病引起的心肌病的小分子药物。这家生物技术公司声称其药物比辉瑞上市的药物更好的稳定剂。2024年，辉瑞报告其Vyndaqel系列TTR稳定剂收入为33亿美元，相比2023年增长了36%。

The

The

FDA approved the BridgeBio TTR drug this past November

FDA于去年11月批准了BridgeBio的TTR药物

and it is marketed in the U.S. under the brand name Attruby. In a prescriptions update included in its report of fourth quarter and full year

并且在美国以品牌名Attruby销售。在其第四季度和全年报告中包含的处方更新中

2024 financial results

2024年财务业绩

, BridgeBio said that as of Feb. 17, 1,028 unique patient prescriptions for the drug have been written by 516 unique prescribers since the U.S. approval. That’s an increase from what the company reported last month during the annual J.P. Morgan Healthcare Conference last month, which Leerink Partners said indicates a broadening prescriber base..

，BridgeBio 表示，截至 2 月 17 日，自美国批准以来，已有 516 名不同的开处方者为该药物开出了 1,028 张不同的患者处方。这比该公司上个月在年度摩根大通医疗保健会议上报告的数字有所增加，Leerink Partners 表示，这表明开处方者的基础正在扩大。

presented by

由...呈现

Artificial Intelligence

人工智能

What Keeps Healthcare CIOs Up at Night: Balancing Technology Investments with Consumer Expectations

什么让医疗行业的首席信息官夜不能寐：在技术投资与消费者期望之间取得平衡

When it comes to managing inbound phone calls, underperformance has devastating cost implications.

当谈到管理呼入电话时，表现不佳会带来毁灭性的成本影响。

By Stephanie Baum

作者：斯蒂芬妮·鲍姆

In Europe, the BridgeBio drug

在欧洲，BridgeBio药物

will be commercialized by Bayer

将由拜耳商业化

per terms of an agreement signed last year. BridgeBio is in line to receive milestone payments and royalties from sales. The first payment is $75 million for the European approval.

根据去年签署的协议条款，BridgeBio将获得里程碑付款和销售版税。第一笔款项是7500万美元，用于欧洲的批准。

Here’s a recap of other recent regulatory developments:

以下是其他近期监管动态的简要回顾：

More Rare Disease Drug Approvals

更多罕见病药物获批

—The FDA

—美国食品药品监督管理局

approved Mirum Pharmaceuticals drug chenodiol

批准了Mirum制药公司的药物chenodiol

, brand name Ctexli, for treating rare lipid storage disease cerebrotendinous xathomatosis (CTX).

，商品名为Ctexli，用于治疗罕见的脂质储存疾病脑腱黄色瘤病（CTX）。

This genetic metabolic disorder results in deficient levels of an enzyme important for breaking down fats. Chenodiol is a naturally occurring human bile acid. Under the brand name Chenodal, chenodiol has been marketed for treating gallstones, but it has been used off label as a treatment for CTX. The new FDA approval makes Ctexli the first FDA-approved treatment for CTX.

这种遗传性代谢紊乱导致一种用于分解脂肪的重要酶水平不足。鹅去氧胆酸是一种天然存在的人类胆汁酸。在品牌名Chenodal下，鹅去氧胆酸已被用于治疗胆结石，但它也被非标签用于治疗CTX。新的FDA批准使Ctexli成为首个FDA批准的CTX治疗方法。

Mirum acquired the drug from Travere Therapeutics in 2023..

Mirum于2023年从Travere Therapeutics收购了该药物。

Healthcare

医疗保健

Using Data to Help Healthcare Practices Succeed

利用数据帮助医疗实践取得成功

A new report from Relatient, A Data-Driven Guide to Patient Access Succes, highlights how focusing on data accuracy and relevance can enhance the performance of healthcare practices.

Relatient的一份新报告《以数据为导向的患者访问成功指南》强调了如何通过关注数据的准确性和相关性来提升医疗实践的绩效。

By Relatient

由Relatient发布

—The

—这

European Commission approved Livdelzi

欧盟委员会批准了Livdelzi

, a Gilead Sciences drug developed for the rare liver disease primary biliary cholangitis (PBC).

，这是吉利德科学公司开发的一种用于治疗罕见肝病原发性胆汁性胆管炎 (PBC) 的药物。

The conditional marketing authorization covers use of the daily pill in combination with ursodeoxycholic acid, an older drug that is the standard PBC treatment. The European decision for Livdelzi follows

有条件营销授权涵盖了每日服用该药丸与熊去氧胆酸（一种作为PBC标准治疗的较老药物）联合使用的情况。欧洲对Livdelzi的决定紧随其后。

FDA approval of the drug last August

去年八月该药物获得FDA批准

. Livdelzi comes from

. Livdelzi 来自

Gilead’s $4.3 billion acquisition of Cymabay Therapeutics

吉利德以43亿美元收购Cymabay Therapeutics公司

last year.

去年。

—The FDA

—美国食品药品监督管理局

approved SpringWorks Therapeutics drug Gomekli

批准了SpringWorks Therapeutics公司的Gomekli药物

as a treatment for tumors caused by the rare genetic disorder neurofibromatosis type 1 (NF1).

作为一种治疗由罕见遗传病神经纤维瘤病1型（NF1）引起的肿瘤的方法。

The approval covers treatment of these tumors in both adults and children. That’s an advantage over AstraZeneca’s Koselugo, which is only approved for treating pediatric NF1 patients.

该批准涵盖了成人和儿童的这些肿瘤治疗。这相比阿斯利康的Koselugo具有优势，后者仅被批准用于治疗儿科NF1患者。

—CSL’s garadacimab received

—CSL的garadacimab获得

approvals in Europe

欧洲的批准

and

和

Japan

日本

for treating adults and adolescents age 12 and older who have hereditary angioedema (HAE).

用于治疗12岁及以上患有遗传性血管性水肿（HAE）的成人和青少年。

The rare disease leads to swelling attacks in tissue beneath the skin. This swelling can close off the airway, putting a patient at risk of death. CSL’s drug, brand name Andembry, is an antibody engineered to inhibit activated Factor XII, a protein that starts the cascade of events leading to unpredictable HAE swelling attacks.

这种罕见病会导致皮下组织发生肿胀发作。这种肿胀可能会阻塞气道，使患者面临死亡风险。CSL公司的药物，商品名为Andembry，是一种经过设计的抗体，旨在抑制活化的XII因子，这是一种引发不可预测的HAE肿胀发作的蛋白质。

Andembry is still under regulatory review in the U.S., Canada, and Switzerland..

Andembry 仍在接受美国、加拿大和瑞士的监管审查。

—Evrysdi, a drug marketed by Roche for the rare disease spinal muscular atrophy, is now

——罗氏公司销售的用于罕见病脊髓性肌萎缩症的药物Evrysdi，现在

approved in a tablet formulation

批准了片剂配方

.

。

The product, which was initially developed by PTC Therapeutics and later partnered with Roche, was

该产品最初由PTC Therapeutics开发，后来与罗氏合作，

first approved

首次批准

in 2020 as a liquid administered via an oral syringe or through a feeding tube. The liquid formulation must be refrigerated and kept away from light. The new Evrysdi tablet offers patients a simpler dosing option that can be stored at room temperature.

在 2020 年，液体通过口腔注射器或通过喂食管给药。液体配方必须冷藏并避光保存。新的 Evrysdi 片剂为患者提供了一种更简单的剂量选择，可以在室温下保存。

Cancer Drug Approvals

癌症药物审批

—Merck’s Welireg is now

—默克公司的Welireg现在

approved in Europe

欧洲批准

as a treatment for adults with von Hillel-Lindau (VHL) disease, a rare genetic disorder that causes benign tumors in the body that can become cancerous.

作为一种治疗成人冯·希佩尔-林道（VHL）病的方法，这是一种罕见的遗传性疾病，会导致身体出现可能癌变的良性肿瘤。

The European Commission’s conditional approval covers patients whose VHL is unsuitable for surgery and requires therapy for renal cell carcinoma, central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors. The commission’s decision also covers the treatment of adults with advanced clear cell renal cell carcinoma that has progressed after two or more lines of therapy.

欧洲委员会的有条件批准涵盖了那些VHL不适合手术且需要治疗肾细胞癌、中枢神经系统血管母细胞瘤或胰腺神经内分泌肿瘤的患者。该委员会的决定还涵盖了对接受过两线或多线治疗后病情进展的晚期透明细胞肾细胞癌成人患者的治疗。

Welireg is a once-daily pill designed to block HIF-2 alpha, inhibiting this protein’s ability to contribute to cancer progression. .

Welireg是一种每日一次的药丸，旨在阻断HIF-2α，抑制该蛋白促进癌症进展的能力。

The FDA approved Welireg in 2021

FDA于2021年批准了Welireg

.

。

—The

—这

FDA approved Romvimza, an Ono Pharmaceutical drug

FDA批准了小野制药的药物Romvimza

for tenosynovial giant cell tumor (TGCT).

用于腱鞘巨细胞瘤（TGCT）。

The drug, which came to Ono via the

这种药物通过Ono公司来到了市场上，

2024 acquisition of its developer, Deciphera Pharmaceuticals

2024年收购其开发商Deciphera Pharmaceuticals

, offers more convenient dosing and a lower risk of liver toxicity compared to Turalio, a Daiichi Sankyo drug that was

，与第一三共制药公司生产的Turalio相比，提供了更方便的剂量和更低的肝毒性风险，该药物是

the first approved systemic therapy

首个获批的系统性疗法

for TGCT.

适用于TGCT。

—Datroway, a TROP-2-targeting antibody drug conjugate (ADC) from partners AstraZeneca and Daiichi Sankyo, landed

—Datroway，由阿斯利康和第一三共合作开发的靶向TROP-2的抗体药物偶联物（ADC），已上市

FDA approval for treating advanced and unresectable breast cancer

FDA 批准用于治疗晚期和不可切除的乳腺癌

that’s classified as HR positive and HER2 negative. It will compete with

这类分类为HR阳性、HER2阴性。它将与

Trodelvy, Gilead Sciences’ TROP-2-targeting ADC

Trodelvy，吉利德科学公司的TROP-2靶向ADC

is already approved in the same indication.

在相同适应症中已获批。

—Axsome Therapeutics’ Symbravo won FDA approval for

—Axsome Therapeutics的Symbravo获得FDA批准

acute treatment of migraine

偏头痛的急性治疗

with or without aura in adults.

有或无先兆的成人。

The tablet pairs two compounds that target multiple pathways behind a migraine attack. Symbravo is made with proprietary Axsome technology that enables the drug to more quickly achieve maximum concentration in the blood and to maintain a long half-life. Symbravo’s label carries a black box warning for cardiovascular and gastrointestinal risks.

这种片剂结合了两种化合物，针对偏头痛发作的多种途径。Symbravo采用专有的Axsome技术制造，能使药物更快达到血液中的最大浓度，并保持较长的半衰期。Symbravo的标签上带有心血管和胃肠道风险的黑框警告。

The approval marks a comeback for the drug, which received an .

批准标志着这种药物的回归，它曾获得过 。

FDA complete response letter in 2022

2022年FDA完整回应信

due to manufacturing issues.

由于制造问题。

—Foundation Medicine received FDA approval for FoundationOne CDx as a

—Foundation Medicine的FoundationOne CDx获得了FDA的批准，作为

companion diagnostic

伴随诊断

for the brain cancer drug Ojemda.

针对脑癌药物Ojemda。

The test detects the BRAF V600 mutation addressed by the drug, developed by Day One Biotherapeutics. The FDA

该测试检测由Day One Biotherapeutics公司开发的药物针对的BRAF V600突变。美国食品药品监督管理局（FDA）

approved Ojemda last April as a treatment for advanced cases of pediatric low-grade glioma

去年四月批准Ojemda用于治疗小儿低级别胶质瘤晚期病例

.

。

—Adcetris, an antibody drug conjugate (ADC) developed by Pfizer subsidiary Seagen,

—Adcetris，一种由辉瑞子公司Seagen开发的抗体药物偶联物（ADC），

added to its label

添加到其标签

the treatment of advanced cases of diffuse large B-cell lymphoma (DLBCL).

晚期弥漫性大B细胞淋巴瘤（DLBCL）的治疗。

The

The

new approval

新批准

covers use of the drug in combination with the cancer drugs Revlimid and Rituxan. The new FDA approval is based on Phase 3 results showing the Adcetris regimen led to a 37% reduction in risk of death compared to Revlimid, Rituxan, and a placebo.

涵盖了该药物与癌症药物Revlimid和Rituxan联合使用的情况。新的FDA批准是基于第三阶段试验结果，显示Adcetris方案相比Revlimid、Rituxan和安慰剂，将死亡风险降低了37%。

Adcetris was first approved in 2011 for Hodgkin lymphoma and systemic anaplastic large cell lymphoma. The drug now has eight FDA approvals in cancer. Pfizer has rights to the drug in the U.S. and Canada; Takeda Pharmaceutical has rights to the product in the rest of the world.

Adcetris于2011年首次获批用于霍奇金淋巴瘤和系统性间变性大细胞淋巴瘤。该药物现在已有八项FDA批准的适应症。辉瑞拥有该药物在美国和加拿大的权利；武田制药则拥有该产品在世界其他地区的权利。

—Blockbuster AstraZeneca cancer drug Calquence, first approved in 2017 for treating advanced mantle cell lymphoma after at least one prior line of therapy, is

—阿斯利康的重磅抗癌药物Calquence，最初于2017年获批用于治疗至少经过一种先前疗法的晚期套细胞淋巴瘤，

now approved as a first-line treatment

现已批准作为一线治疗

for this rare and aggressive blood cancer

对于这种罕见且侵袭性的血液癌症

. The drug, a BTK inhibitor, is the first in its class approved as a first-line MCL treatment.

这种药物是一种BTK抑制剂，是其同类中首个被批准作为一线MCL治疗的药物。

—Amgen cancer drug Lumakras

—安进公司的抗癌药物Lumakras

expanded its FDA approval

扩大了其FDA批准范围

to metastatic colorectal cancer that is positive for the KRAS G12c mutation and is also unresponsive or resistant to chemotherapy. The regulatory nod in this indication covers use of the daily pill in combination with Vectibix, an Amgen antibody drug approved for colorectal cancer.

针对KRAS G12c突变阳性且对化疗无反应或耐药的转移性结直肠癌。该适应症的监管批准涵盖了每日口服药物与安进公司抗体药物Vectibix（已获批用于结直肠癌）的联合使用。

Lumakras was initially approved in 2022 as a treatment for non-small cell lung cancer

Lumakras 于2022年首次获批用于治疗非小细胞肺癌。

driven by the KRAS G12C mutation.

由KRAS G12C突变驱动。

Approvals in Immunology

免疫学批准

—Galderma drug Nemluvio received

—Galderma药物Nemluvio已收到

European Commission approval

欧盟委员会批准

as a treatment for the inflammatory skin disorders atopic dermatitis and prurigo nodularis.

作为一种治疗炎症性皮肤病（如特应性皮炎和结节性瘙痒症）的方法。

The once-monthly injection is an antibody designed to block IL-31 signaling. The

每月一次的注射是一种旨在阻断IL-31信号传导的抗体。

FDA approved Nemluvio

FDA 批准了 Nemluvio

for prurigo nodularis and atopic dermatitis last year.

去年用于治疗结节性瘙痒和特应性皮炎。

—Eli Lilly’s Omvoh, a biologic drug

—礼来公司的Omvoh，一种生物药物

first approved in 2023

首次获批于2023年

as a treatment for ulcerative colitis,

作为治疗溃疡性结肠炎的方法，

added moderately to severely active Crohn’s disease

中度至重度活动性克罗恩病

to its label.

给它的标签。

The drug is an antibody that targets IL-23p19, a protein that contributes to gastrointestinal inflammation. Regulatory submissions in Crohn’s are currently under review in Europe and Japan. In ulcerative colitis, the drug is approved in 44 countries.

该药物是一种针对 IL-23p19 的抗体，这种蛋白质会导致胃肠道炎症。目前，克罗恩病的监管提交正在欧洲和日本接受审查。在溃疡性结肠炎方面，该药物已在 44 个国家获得批准。

—Palforzia, a peanut allergy immunotherapy marketed by Stallergenes Greer, expanded the product’s approved uses in Europe to include the treatment of

—Stallergenes Greer公司销售的花生过敏免疫疗法Palforzia，扩大了该产品在欧洲的批准用途，包括治疗

children ages 1 through 3

1至3岁的儿童

.

。

The initial approval for Palforzia covered children age 4 through 17. The new approval in Europe comes about six months after the therapy secured a similar

Palforzia最初的批准适用于4至17岁的儿童。该疗法在欧洲获得新批准，大约是在其获得类似批准的六个月后。

regulatory decision from the FDA

FDA的监管决定

. Privately held

. 私人持有

Stallergenes Greer added Palforzia to its allergy products portfolio via a 2023 deal with Nestlé

斯塔勒吉尼斯·格里尔公司通过2023年与雀巢的交易，将Palforzia添加到其过敏产品组合中。

.

。

Approvals for Neuro, Pain Meds

神经、止痛药的审批

—Supernus Pharmaceuticals received

—Supernus Pharmaceuticals收到

FDA approval for Onapgo, a drug/device combination product

FDA 批准了 Onapgo，一种药物/设备组合产品

that continuously infuses the drug apomorphine to treat the “off” episodes experienced by Parkinson’s disease patients.

持续输注药物阿扑吗啡，用于治疗帕金森病患者的“关闭”期。

Onapgo joins a Supernus Parkinson’s drug lineup that includes Apokyn, an injection pen product that administers apomorphine, and Gocovri, a capsule approved to treat dyskinesia and off time in Parkinson’s patients.

Onapgo加入了一个Supernus帕金森病药物系列，该系列包括注射笔产品Apokyn（可递送阿扑吗啡）和胶囊Gocovri（获批用于治疗帕金森病患者的运动障碍和“关闭”时间）。

—

—

Vertex Pharmaceuticals’ non-opioid drug Journavx won FDA approval

Vertex制药公司的非阿片类药物Journavx获得FDA批准

as a first-in-class treatment for moderate-to-severe acute pain in adults.

作为治疗成人中度至重度急性疼痛的首创新疗法。

The twice-daily pill targets NaV1.8, a pathway in the peripheral nervous system. By stopping pain signals in the periphery before they reach the brain, drugs in this class are intended to avoid the addiction risks poised by opioids, which hit targets in the brain.

这种每日服用两次的药片针对外周神经系统中的NaV1.8通路。通过在外周阻止疼痛信号到达大脑之前将其拦截，这类药物旨在避免阿片类药物所带来的成瘾风险，因为阿片类药物作用于大脑中的靶点。

—The FDA

—美国食品药品监督管理局

approved Johnson & Johnson’s Spravato

批准了强生公司的Spravato

as a monotherapy for adults with treatment-resistant depression.

作为治疗成人难治性抑郁症的单一疗法。

The nasal spray drug’s initial approval in 2019 covered its use in combination with an oral antidepressant for treatment-resistant depression in adults.

2019年，该鼻喷雾药物最初获批，可与口服抗抑郁药联合使用，用于治疗成人难治性抑郁症。

Vaccine Approvals

疫苗审批

—Bavarian Nordic received FDA approval for its

— Bavarian Nordic 获得了 FDA 的批准

virus-like particle chikungunya vaccine

类病毒颗粒基孔肯雅疫苗

, which will be marketed under the brand name Vimkunya.

，该药物将使用品牌名称Vimkunya进行销售。

The FDA nod covers use of the vaccine in those age 12 and older. That’s a broader range than

美国食品药品监督管理局的批准涵盖了12岁及以上人群使用该疫苗。这一范围比

Valneva’s Ixchiq

瓦尔内瓦的Ixchiq

, which became the first FDA-approved chikungunya vaccine in 2023. Ixchiq is indicated for use in those age 18 and older. Bavarian Nordic’s new approval was accompanied by a tropical disease priority review voucher, which the company said it plans to sell.

，该疫苗在2023年成为首个获得FDA批准的基孔肯雅热疫苗。Ixchiq适用于18岁及以上人群。Bavarian Nordic公司的这项新批准还附带了一张热带病优先审查券，该公司表示计划将其出售。

—A GSK vaccine covering five groups of bacteria that cause meningococcal disease is now

—GSK公司生产的一种覆盖五组引起脑膜炎球菌病的细菌的疫苗现已

FDA approved

FDA批准

for use in those ages 10 through 25.

用于10至25岁的人群。

The vaccine, brand name Penmenvy, combines components of two already available GSK meningococcal vaccines, Bexsero and Menveo. Penmenvy will now compete with

该疫苗品牌名称为Penmenvy，结合了GSK已有的两种脑膜炎球菌疫苗Bexsero和Menveo的成分。Penmenvy现在将与

Pfizer’s Penbraya, which also covers five bacterial groups

辉瑞的Penbraya，也涵盖了五种细菌群

. Both vaccines are administered as two intramuscular injections given six months apart.

两种疫苗均通过肌肉注射接种，两次注射间隔六个月。

—A Covid-19 vaccine developed by partners Arcturus Therapeutics and CSL is now

—Arcturus Therapeutics和CSL合作开发的新冠疫苗现已

approved in Europe

欧洲批准

.

。

The vaccine, brand name Kostaive, employs self-amplifying RNA. Unlike traditional mRNA vaccines, self-amplifying RNA vaccines instruct the body to make more mRNA and protein to boost the immune response.

这种疫苗的品牌名称为Kostaive，采用了自我扩增型RNA。与传统的mRNA疫苗不同，自我扩增型RNA疫苗指导身体产生更多的mRNA和蛋白质，以增强免疫反应。

Kostaive was originally developed by Arcturus. In 2022, CSL paid $200 million up front to collaborate on the vaccine. Per deal terms, Arcturus is responsible for regulatory filings in the U.S. and Europe as well as R&D of next-generation vaccine candidates. CSL subsidiary Seqirus takes the lead on all other R&D in Covid-19, influenza, and other fields..

科斯塔伊夫最初由阿克图勒斯公司开发。2022年，CSL支付了2亿美元预付款以合作开发该疫苗。根据协议条款，阿克图勒斯负责在美国和欧洲的监管申报以及下一代候选疫苗的研发。CSL子公司Seqirus则主导所有其他关于新冠、流感及其他领域的研发工作。

More Regulatory Approvals

更多监管批准

—Izervay, a geographic atrophy drug marketed by Astellas Pharma, is now

—Izervay，安斯泰来制药公司销售的一种地理性萎缩药物，现在

approved for longer dosing

批准更长的给药间隔

.

。

The product’s initial 2023 approval permitted the once-monthly eye injection to be

该产品在2023年的最初批准允许每月一次的眼部注射

administered for up to 12 months

施用长达12个月

. The new approval removes the 12 month restriction. However, the Astellas product did not receive the FDA O.K. for every-other-month administration. That additional flexibility would have put it on par with Apellis Pharmaceuticals’ geographic atrophy drug

新批准取消了12个月的限制。然而，Astellas的产品并未获得FDA每两个月给药一次的批准。这一额外的灵活性将使其与Apellis Pharmaceuticals的地理萎缩药物相当。

Syfovre, which is approved for monthly and every-other-month dosing

Syfovre，获批用于每月和每两个月一次的给药方案

.

。

—Novo Nordisk drug

—诺和诺德药物

Ozempic is now approved for treating chronic kidney disease

Ozempic现已获批用于治疗慢性肾病

in patients with type 2 diabetes.

在2型糖尿病患者中。

The blockbuster drug was initially approved for type 2 diabetes, which is a risk factor for chronic kidney disease. In the pivotal study supporting the latest regulatory nod, Ozempic led to a

这种重磅药物最初是为2型糖尿病获批的，而2型糖尿病是慢性肾病的一个风险因素。在支持最新监管批准的关键研究中，Ozempic导致了

24% reduction in kidney disease complications

肾病并发症减少24%

compared to a placebo.

与安慰剂相比。

—AbbVie’s pairing of the antibiotics aztreonam and avibactam received

—AbbVie公司将抗生素氨曲南和阿维巴坦配对使用获得了

FDA approval

FDA批准

for the treatment of complicated intra-abdominal infections,

用于治疗复杂的腹腔内感染，

giving clinicians another tool to address antimicrobial resistance. The therapy, which will be marketed under the brand name Emblaveo, is indicated for use in combination with Flagyl, a Pfizer antibiotic. Emblaveo was developed in partnership with Pfizer. AbbVie has commercialization rights to the drug in the U.S and Canada; Pfizer is responsible for the antibiotic’s commercialization in the rest of the world..

为临床医生提供了另一种应对抗菌素耐药性的工具。该疗法将以Emblaveo品牌上市，与辉瑞的抗生素Flagyl联合使用。Emblaveo由辉瑞合作开发，艾伯维拥有该药物在美国和加拿大的商业化权利；辉瑞负责该抗生素在世界其他地区的商业化。

Clinical Holds and Regulatory Setbacks

临床暂停和监管挫折

—The FDA sent Harmony Biosciences a

—美国食品药品监督管理局（FDA）向Harmony Biosciences发送了

refuse to file letter

拒绝归档信件

for pitolistant, a drug the biotech is seeking to commercialize as a treatment for excessive daytime sleepiness in adults who have idiopathic hypersomnia.

对于pitolistant，这家生物技术公司正寻求将其商业化，用于治疗患有特发性嗜睡症的成人白天过度嗜睡。

In a Phase 3 withdrawal study, the once-daily tablet did not achieve statistical significance compared to placebo. The biotech said a Phase 3 registrational study in idiopathic hypersomnia is on track to begin in the fourth quarter of this year. Pitolistant is already available as a treatment for excessive daytime sleepiness or cataplexy in adults with narcolepsy, marketed as Wakix in this indication..

在一项三期撤药研究中，这种每日一次的片剂与安慰剂相比未达到统计学显著性。这家生物技术公司表示，针对特发性嗜睡症的三期注册研究有望在今年第四季度开始。Pitolistant 已作为治疗成人嗜睡症患者的日间过度嗜睡或猝倒的药物上市，该适应症的商品名为 Wakix。

—Moderna’s Phase 3 test of its two-season norovirus vaccine candidate, mRNA-1403, is under an

—Moderna的两季诺如病毒疫苗候选物mRNA-1403的III期试验正在进行中，

FDA clinical hold

FDA临床暂停

.

。

The company said the clinical trial halt is due to a single report of Guillain- Barré syndrome, nerve damage that can be a complication of vaccines. The adverse event is under investigation. The company does not expect the clinical hold will affect the trial readout timeline in the northern hemisphere, where the study is already fully enrolled.

公司表示，临床试验暂停是由于单一的吉兰-巴雷综合征报告，这是一种可能由疫苗引发的神经损伤并发症。该不良事件正在调查中。公司预计临床暂停不会影响北半球的试验结果时间表，因为该研究已经在该地区完成了全部招募。

The timing of a data readout will depend on case accruals. Second season enrollment is being prepared in the southern hemisphere..

数据读取的时间将取决于病例的积累。南半球正在准备第二季节的注册。

—The FDA placed a

—美国食品药品监督管理局（FDA）放置了一个

clinical hold

临床试验暂停

on Ebvallo, a treatment for advanced cases of Epstein-Barr virus positive post-transplant lymphoproliferative disease.

在Ebvallo上，这是一种用于治疗移植后Epstein-Barr病毒阳性晚期淋巴增殖性疾病的药物。

The hold came days after the

持仓发生在几天之后

FDA rejected

FDA拒绝了

the immunotherapy due to findings from an inspection of a third-party manufacturing facility. The company said the Ebvallo clinical hold relates to the issues flagged in the complete response letter. Ebvallo, which is made by engineering T cells from healthy donors, was

由于对第三方生产设施进行检查后发现的问题，免疫疗法被叫停。公司表示，Ebvallo的临床暂停与完整回应函中指出的问题有关。Ebvallo是通过工程化健康捐赠者的T细胞制成的，

approved in Europe in 2022

2022年在欧洲获批

.

。

Photo: sinonimas, Getty Images

照片来源：sinonimas，Getty Images

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