A Randomised Crossover Pilot Study of the Effects of Tezacaftor/Ivacaftor and Ivacaftor on Gastrointestinal Function Using Magnetic Resonance Imaging Parameters in People With Cystic Fibrosis

People with Cystic Fibrosis (CF) have problems digesting their food properly. More than 8 in 10 people with CF must take medication to assist their digestion. In spite of this, complications such as bowel blockage occur.
Finding out how already licenced drugs for CF work in the gut is the first step in repurposing medications. Tezacaftor/Ivacaftor with Ivacaftor is a drug combination which corrects the basic defect in CF an has shown improvements on lung function.
The purpose of this study is to evaluate, using Magnetic Resonance Imaging (MRI) and patient-reported outcomes, whether Tezacaftor/Ivacaftor with Ivacaftor has an effect on improving gastrointestinal problems in CF.

开始日期2019-09-03

申办/合作机构

Nottingham University Hospitals Nhs Trust

[+5]

NCT03587961

/ Recruiting早期临床1期IIT

Personalized Theratyping Trial

The purpose of this study is to explore the use of off-label CFTR modulators that may affect CFTR function in patients with CFTR mutations that are not currently approved for these drugs.

开始日期2019-08-01

申办/合作机构

The University of Alabama at Birmingham

100 项与替扎卡托/依伐卡托相关的临床结果

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100 项与替扎卡托/依伐卡托相关的转化医学

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100 项与替扎卡托/依伐卡托相关的专利（医药）

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项与替扎卡托/依伐卡托相关的文献（医药）

2024-10-01·Journal of Cystic Fibrosis

Development of metabolic syndrome in people with Cystic Fibrosis one year after exposure to elexacaftor-tezacaftor-ivacaftor

Article

作者: Finklea, James D ; Reisch, Joan ; Mirfakhraee, Sasan ; Ratti, Gregory A. ; Jain, Raksha ; Finklea, James D. ; Cohen, Leah ; Ratti, Gregory A ; Smith, Hannah

BACKGROUND:

The constellation of hypertension, truncal obesity, impaired fasting glucose, low high-density lipoprotein, and hypertriglyceridemia is known as metabolic syndrome (MetSyn) and is associated with cardiovascular and other diseases. Elexacaftor-tezacaftor-ivacaftor (ETI) in people with cystic fibrosis (pwCF) is associated with weight gain but effects on cardiovascular risk are unknown. This study sought to investigate ETI exposure and risk for development of MetSyn in pwCF.

METHODS:

A prospective cohort study including pwCF ≥ 18 years old exposed to ETI was performed. All data for calculating MetSyn was collected from the electronic medical record at initiation and 1 year ± 3 months after starting ETI. A total of 152 pwCF exposed to ETI and 34 pwCF never exposed to CF transmembrane conductance regulator modulators were included in the analysis. Changes to hypertension classification was also examined over this period.

RESULTS:

After 1 year of ETI there was an increase in MetSyn from 13 to 30 pwCF, p < 0.0001. No new cases of MetSyn were seen in the group not exposed to ETI. After 1 year of ETI, more people met criteria for class 1 (BP 130-139/90-99 mm Hg) or class 2 hypertension (BP ≥140/≥90 mm Hg) regardless of prior modulator exposure, p < 0.0001.

CONCLUSIONS:

Exposure to ETI for 1 year resulted in an increased number of cases of MetSyn. There was an increased incidence of hypertension associated with ETI exposure. Additional studies are needed to further examine this trend and to determine if these changes will translate to cardiovascular complications over time.

2024-02-14·mBio

Longitudinal profiling of the intestinal microbiome in children with cystic fibrosis treated with elexacaftor-tezacaftor-ivacaftor

Article

作者: Hadjifrangiskou, Maria ; Brown, Rebekah F. ; Nicholson, Maribeth R. ; Waalkes, Adam ; Reasoner, Seth A. ; Edwards, Kathryn M. ; Penewit, Kelsi ; Bernard, Rachel ; Sokolow, Andrew G. ; Lewis, Janessa ; Salipante, Stephen J.

Cystic fibrosis (CF) is an autosomal recessive disease with significant gastrointestinal symptoms in addition to pulmonary complications. Recently approved treatments for CF, CF transmembrane conductance regulator (CFTR) modulators, are anticipated to substantially improve the care of people with CF and extend their lifespans. Prior work has shown that the intestinal microbiome correlates with health outcomes in CF, particularly in children. Here, we study the intestinal microbiome of children with CF before and after the CFTR modulator, ELX/TEZ/IVA. We identify promising improvements in microbiome diversity, reduced measures of intestinal inflammation, and reduced antibiotic resistance genes. We present specific bacterial taxa and protein groups which change following ELX/TEZ/IVA. These results will inform future mechanistic studies to understand the microbial improvements associated with CFTR modulator treatment. This study demonstrates how the microbiome can change in response to a targeted medication that corrects a genetic disease.

2024-01-01·ERJ open research

Impact of CFTR modulators on exercise capacity in adolescents with cystic fibrosis

Article

作者: Gaillard, Erol ; Madge, Joe ; Ahmed, Molla Imaduddin ; Blyth, Natalie ; Dayman, Naomi

Background:

Exercise capacity is an independent predictor of clinical worsening in cystic fibrosis (CF). There is limited evidence of the impact of cystic fibrosis transmembrane conductance regulator (CFTR) modulators on exercise capacity in children with CF. The aim of the present study was to assess the impact of CFTR modulators on exercise capacity in a cohort of adolescents with CF.

Methods:

A prospective single-centre cohort study was carried out. Cardiopulmonary exercise testing (CPET) was performed at baseline, prior to starting Symkevi or Kaftrio and between 4 and 8 months after starting treatment.

Results:

19 adolescents with CF had CPET performed prior to and after CFTR modulator treatment, between December 2019 and March 2022. Breathing reserve improved in the whole cohort, with greater improvement in the modulator-naïve patients after starting treatment with Kaftrio. There was no improvement in peak oxygen uptake and anaerobic threshold after 4 to 8 months of treatment with CFTR modulators.

Conclusion:

Exercise testing with CPET can be used as an additional tool to monitor response to CFTR modulators. Breathing reserve on CPET may provide a surrogate marker to monitor the improvement in CF lung disease with CFTR modulator treatment.

项与替扎卡托/依伐卡托相关的新闻（医药）

2024-12-19

·PRNewswire

A Success Story: Digital Opinion Leader Analysis for Cystic Fibrosis Treatments | DelveInsight

A case study on how a pharmaceutical company specializing in cystic fibrosis treatments turned to DelveInsight for comprehensive analysis and insights into the digital cystic fibrosis landscape in the UK. DelveInsight identified key Digital Opinion Leaders (DOLs) and synthesized their opinions, enabling the client to strategically leverage these insights for drug development, marketing, and patient advocacy, ensuring a competitive edge in the cystic fibrosis market. LAS VEGAS, Dec. 19, 2024 /PRNewswire/ -- DelveInsight, a leader in healthcare competitive intelligence and consulting company, published a success study on Digital Opinion Leader (DOL) Analysis in the Cystic Fibrosis Market . A pharmaceutical company specializing in cystic fibrosis treatments approached DelveInsight to conduct an in-depth analysis of DOLs in the UK. The client sought to identify key digital influencers and understand their perspectives on cystic fibrosis treatments, leveraging these insights to inform drug development, marketing strategies, and patient advocacy efforts. Cystic fibrosis is a life-limiting autosomal recessive genetic disorder that primarily affects the lungs and digestive system, caused by mutations in the CFTR gene. According to DelveInsight's latest Cystic Fibrosis Market and Epidem Forecast Report, the estimated total diagnosed prevalent cases of cystic fibrosis in the 7MM in 2023 were ~68K cases. The UK had the highest number of diagnosed cystic fibrosis cases, with nearly 11K, followed by France with 7.5K and Germany with 7K. In the EU4 and the UK, the cystic fibrosis market size was nearly USD 7 billion, accounting for almost 39.4% of the total 7MM market size in 2023. This is expected to increase significantly by 2034. Download the Sample Report to Explore Future Trends in the Cystic Fibrosis Market In the field of cystic fibrosis, DOLs play a significant role in shaping perceptions and influencing the direction of treatment, policy, and patient care. These are influential healthcare professionals or researchers who have an active online presence and contribute to discussions, research, and advocacy. For pharmaceutical companies developing cystic fibrosis treatments, understanding the DOL landscape is crucial for gaining insights into market dynamics and stakeholder perceptions and identifying strategic opportunities for drug development, marketing, and patient advocacy. Several medications have been approved to manage cystic fibrosis, with therapies targeting the CFTR protein to correct its defects. One of the most groundbreaking treatments is TRIKAFTA, a combination of elexacaftor, tezacaftor, and ivacaftor, which has revolutionized cystic fibrosis treatment by improving lung function in patients with specific genetic mutations. Other notable drugs include SYMDEKO (tezacaftor/ivacaftor) and ORKAMBI (lumacaftor/ivacaftor), which also target the CFTR protein. These therapies aim to address the root cause of the disease, improving symptoms and quality of life for patients. However, despite these advancements, the current treatment landscape still faces challenges, such as variability in treatment efficacy and accessibility to newer therapies for certain patient populations. The pipeline for cystic fibrosis treatments continues to evolve, with several promising drugs in the cystic fibrosis pipeline. Ensifentrine (Verona Pharmaceuticals) is a dual inhibitor of the enzymes responsible for inflammation and mucus production in the lungs, showing potential as an add-on therapy for patients with CF. VX-121/TEZ/VX-561 (Vertex Pharmaceuticals) is another exciting treatment, combining triple therapy to enhance CFTR function and improve lung function. LAU-7b (Laurent Pharmaceuticals Inc.) aims to restore CFTR function and reduce inflammation. Lonodelestat (Pari Pharma GmbH) is being investigated for its ability to reduce neutrophil elastase and inflammation in the lungs of CF patients. These drugs, if successful, could significantly improve treatment options and outcomes for CF patients. Several companies are at the forefront of developing treatments for cystic fibrosis. Eloxx Pharmaceuticals, Inc. focuses on treatments that aim to restore protein function in CF patients with specific genetic mutations. Vertex Pharmaceuticals is a leader in the CF market, with therapies like TRIKAFTA and ongoing pipeline developments. Santhera Pharmaceuticals is exploring potential treatments to address pulmonary issues in CF patients. DelveInsight helped the client by curating insights from the digital cystic fibrosis landscape and analyzing the opinions of key digital influencers. By identifying and analyzing these DOLs, DelveInsight provided actionable insights and strategic recommendations for the client. The company gained a deeper understanding of the market dynamics, stakeholder perceptions, and the evolving digital landscape surrounding cystic fibrosis. These insights were invaluable for informing drug development strategies, understanding the gaps, patient burden,, and patient advocacy initiatives. Armed with comprehensive information on DOLs and their impact, the client was better equipped to make informed decisions, enhance patient engagement, and improve the overall market approach for cystic fibrosis treatments. Our client approached us with multiple objectives: Understand the DOL Landscape in the UK for cystic fibrosis. Identify Key Digital Opinion Leaders influencing discussions about cystic fibrosis. Analyze the Opinions of DOLs on treatment options and emerging therapies. Leverage Insights to refine drug development, marketing strategies, and patient advocacy efforts. Outcome of DelveInsight's Digital Opinion Leader Analysis: We identified and profiled key digital opinion leaders (DOLs) in the UK cystic fibrosis community, focusing on those with strong online presence, high engagement, and CF expertise. Through content analysis, we gained insights into opinions, trends, and key areas like treatment, patient care, advocacy, and research within the CF community. We mapped the CF digital ecosystem, highlighting connections and interactions between DOLs and stakeholders revealing network dynamics and engagement patterns. We assessed the impact of DOLs using metrics like follower count, engagement rate, and content virality, helping clients prioritize engagement strategies and collaborations. DelveInsight specializes in comprehensive Key Opinion Leader (KOL) and Digital Opinion Leader (DOL) opinion analysis, offering deep insights into industry trends, expert perspectives, and influencer dynamics. Our expertise enables clients to identify and understand the nerve of the trends by providing analysis through the most impactful thought leaders and digital influencers. By leveraging our tailored strategies and data-driven approaches, we help businesses strengthen their approach, refine decision-making, and amplify their market impact. Connect with us today to explore how we can support your specific needs and discuss opportunities for strategic engagement. Request a Proposal Now ! Why Choose DelveInsight? DelveInsight offers comprehensive expertise in the respiratory disease domain, providing in-depth market research, consulting services, and pipeline analysis tailored to the evolving needs of the industry. With a sharp focus on respiratory conditions, DelveInsight delivers robust reports covering emerging therapies, market trends, competitive landscapes, and unmet needs. DelveInsight's respiratory-specific solutions empower stakeholders to make data-driven decisions, optimize their R&D efforts, and seize growth opportunities in the dynamic and rapidly advancing respiratory disease market. Market Assessment Services in the Genetic Disorders Segment, DelveInsight, provide a 360-degree view of the market landscape. Our expertise includes epidemiology-based market forecasts up to 2034, offering precise insights into current and future market share uptake of emerging therapies. These forecasts, combined with detailed analyses of emerging trends, competitive dynamics of the pipeline therapies, and unmet needs, enable stakeholders to strategize effectively and identify growth opportunities. Whether it's the pipeline analysis, pricing strategies, or competitive benchmarking, DelveInsight delivers actionable intelligence tailored to your goals. Contact us Today to discuss how our services can drive success in your endeavors within the genetic disorder market. Competitive Intelligence Services Tailored to Genetic Disorder Domain: DelveInsight's competitive intelligence services provide real-time, accurate insights across the different therapeutic domains and genetic disorders being one of our fortes, providing detailed insights into advancements across rare and inherited conditions such as cystic fibrosis, Duchenne muscular dystrophy, and sickle cell anemia etc to name a few. Our services analyze competitors' pipelines, clinical trial progress, gene and cell therapy innovations, and market entry strategies, as well as regulatory updates and patent landscapes specific to genetic disorders. This intelligence enables stakeholders to identify emerging threats, uncover growth opportunities, and develop targeted strategies to remain competitive in this evolving therapeutic area. Connect with us today to explore how we can help you succeed in the genetic disorders market. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform , PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

上市批准

2024-11-29

·生物制品圈

2024年市值最高的20大制药公司

据Biospace统计，今年以来，在市值最高的20大制药公司中，有一半的公司股价上涨，有一半的公司股价下跌。其中，礼来年内股价涨幅位居市值最高的20大制药公司榜首。这并不意外，因为其畅销减肥药Zepbound于2023年底获批。同时，礼来似乎巧妙地避开了国会降低药价的持续施压，而其竞争对手诺和诺德则直接遭遇了参议员伯尼·桑德斯（Bernie Sanders）的严厉批评。实际上，今年制药巨头公司股价的最大涨幅背后，都有着畅销药物销售额激增、药物获批振奋人心以及竞争对手失误等引人入胜的故事。而另一方面，股价明显下跌的公司则面临着临床试验失败、市场压力以及专利即将到期等困境。例如，辉瑞因受到激进投资者的压力，年内股价下跌超过10%。而默沙东则因其癌症重磅药物Keytruda的专利即将到期，年内股价下跌超过7%。下面分别挑选了三家股价上涨（礼来、Vertex和诺和诺德）和三家股价下跌（辉瑞、安进和默沙东）的公司，对其股价变动背后的故事进行了回顾。所有市值和股价变动数据均截至美国东部时间本周二下午1点30分。股价为前一日收盘价。三家股价上涨的公司 1、礼来今年可谓是礼来的胜利之年。自2023年12月31日以来，礼来的市值增长了1628亿美元。其股价在9月达到每股约960美元的高点。反映在公司股东们身上，据公司首席执行官大卫·里克斯（David Ricks）在第三季度收益电话会议上表示，他们已获得了超过16亿美元的股息和股票回购。那是什么推动着礼来的股价暴涨呢？主要是肥胖症药物组合。2022年，替尔泊肽首次获得美国食品药品监督管理局（FDA）批准，用于治疗2型糖尿病。2023年11月，FDA进一步批准替尔泊肽作为减肥药物上市，商品名为Zepbound，适用于治疗肥胖（BMI≥30）或超重（BMI≥27，且伴有至少一种体重相关疾病，如高血压、2型糖尿病或高胆固醇）的成年人。礼来表示，截至9月底，Zepbound已在10个国家获得批准，并将在另外12个国家推出。公司管理层告诉Leerink Partners分析师，中国是近期的重点，有可能在2025年推出。然而，礼来再次落后于竞争对手诺和诺德，后者最近在中国推出了其肥胖症治疗药物Wegovy（司美格鲁肽），且在美国市场也享有先发优势。 Leerink Partners表示，礼来股价的上涨空间巨大。随着研究的推进，肥胖症和糖尿病方面的积极消息预计将继续传来，并确认进一步的健康益处。该公司还拥有强大的肥胖症药物研发管线，包括额外的胰高血糖素样肽-1（GLP-1）选项，如口服的orforglipron和另一种注射剂retatrutide，分别预计将在2025年和2026年获得III期结果。此外，礼来还在斥资引进更多研发管线资产，去年与Fauna Bio和Haya Therapeutics达成了许可协议，并收购了Versanis Bio和Morphic Holding。在阿尔茨海默病领域，礼来刚刚获得了疾病修饰治疗药物Kisunla的批准，这使其与渤健（Biogen）和卫材（Eisai）的Leqembi形成了竞争。不过值得注意的是，礼来股价也面临着一些压力，Leerink Partners指出，这些压力包括定价质疑、需求放缓和支付方限制。此外，除了诺和诺德之外，还有许多即将出现的竞争，如安进的肥胖症候选药物MariTide，本周在备受期待的读出结果后，将进入III期试验。 2、Vertex Vertex是囊性纤维化（CF）领域的领军者，其旗下产品包括Trikafta、Kalydeco、Symdeko，以及2023年末获批的Casgevy，且该系列产品的增长潜力似乎仍未触顶。William Blair的分析师对Vertex在第三季度后上调业绩指导表示满意。 Vertex还将开始从与CRISPR Therapeutics合作开发的基因疗法Casgevy中获得收入。患者终于开始接受这种镰状细胞病治疗药物的输注。囊性纤维化系列产品的年收入正从90亿美元向William Blair估计的峰值120亿美元迈进。值得注意的是，2025年对于Vertex而言在监管方面将是至关重要的一年，因为FDA将在1月对囊性纤维化药物vanzacaftor三联疗法（vanza triple）和口服止痛药suzetrigine做出两项决定。这意味着Vertex有望在2025年初推出两款新药。其中，止痛药物可能是最令人兴奋的，因为该市场急需非成瘾且耐受性更好的可选药物。 3、诺和诺德自Wegovy获批以来，诺和诺德的市值在过去两年里大幅增长。今年6月，其市值达到6429亿美元的高点，股价达到每股约127美元的高点，但随后回落至接近年初的106美元左右。诺和诺德在过去一年的股价起伏与围绕GLP-1领域的新闻动态密切相关。周二，受两条利好消息——拜登政府提议为参加医疗保险和医疗补助计划的患者支付减肥药费用、安进的MariTide减肥药数据最终低于预期的下限——的双重影响，诺和诺德的股价上涨。三家股价下跌的公司 1、辉瑞 Leerink Partners本月初在给客户的一份报告中写道，辉瑞正在使其新冠业务“正常化”。但辉瑞的新冠系列药物所经历的一切真的正常吗？也许更好的说法是，辉瑞已经度过了开发首款获批的新冠疫苗Comirnaty并随后推出最广泛使用的治疗药物Paxlovid后的蜜月期。虽然这两种药物仍在使用，但随着疫情的减弱和疫苗接种率的下降，该系列药物已面临重大阻力。 2021年12月，辉瑞的股价达到约60美元的高点。而今年，其股价已下跌9%以上，目前徘徊在26美元左右。股价下跌促使激进投资者Starboard Value浮出水面，推动变革，特别是指责首席执行官阿尔伯特·布尔拉（Albert Bourla）。但辉瑞公布第三季度收益超预期后，这场运动就像它突然出现时一样迅速消退。 Leerink Partners表示，辉瑞今年成功地将Paxlovid从美国政府的市场模式转变为商业市场，后者将依赖疾病爆发而非政府的大宗采购订单。在其他方面，辉瑞有像Eliquis这样的产品推动收入；然而，这种治疗方法是首批进入医疗保险药物价格谈判的药品之一。但Guggenheim分析师瓦米尔·迪万（Vamil Divan）在10月底收益发布后警告称，投资者仍然担心辉瑞整个产品组合中面临的严峻专利悬崖问题。就在本周，另一家竞争对手打击了辉瑞的产品组合，BridgeBio获得了FDA对治疗转甲状腺素淀粉样变性心肌病的Attruby的批准。辉瑞将tafamidis作为Vyndaqel和Vyndamax用于心脏适应症，而Alnylam有Amvuttra，目前获批用于治疗ATTR淀粉样变性，并自本周起接受FDA对ATTR-CM的审查。辉瑞还在RSV疫苗市场与GSK以及Moderna展开激烈竞争。在研发管线的早期阶段，辉瑞预计将在2025年初获得一种名为danuglipron的口服肥胖症候选药物的试验结果。这将是一个决定性的时刻，因为如果成功，辉瑞将能够重新跻身与礼来和诺和诺德并列。 2、安进上周，安进的股价下跌约1.22%，而在周二则出现急剧转折。备受瞩目的II期MariTide研究数据公布，显示减重效果为20%，低于分析师的预期下限。这些数据与同类患者群体中礼来的tirzepatide的III期研究结果相当。这一消息导致该公司股价周二下跌8%。BMO资本市场的埃文·西格曼（Evan Seigerman）曾称即将公布的研究结果是安进股价的“成败关键”。现在看来，情况确实如此。周二收盘时，安进市值蒸发了约75.3亿美元。 Jefferies敦促投资者保持冷静，由于预期和竞争激烈，想要取得明显优势几乎是不可能的。“从一开始，情况就一直充满挑战：肥胖症药物领域一直难以捉摸，其他同行最近也都股价下跌，市场对‘差异化’抱有高度期望，而华尔街总是追求更高的疗效，”Jefferies集团写道。安进预计，随着投资者消化这些数据，安进的股价将缓慢恢复正常。 3、默沙东 Keytruda一直是默沙东的王牌，该药彻底改变了癌症治疗领域，衍生出数十种适应症，且其临床项目仍在不断产出数据，以争取获得更多FDA的批准。但近年来，情况发生了变化。尤其是今年的相关消息——该药在结直肠癌、非小细胞肺癌、皮肤鳞状细胞癌和小细胞肺癌方面的试验失败或中止，可能预示着Keytruda达到了其极限。 HPV疫苗Gardasil则是默沙东的另一张王牌。自7月默沙东高管透露Gardasil在中国发货延迟以来，公司股价就一直在下滑。其首席财务官卡罗琳·利奇菲尔德（Caroline Litchfield）表示，发货放缓可能意味着默沙东在年底前将发送少于全年合同约定的剂量。首席执行官罗伯特·戴维斯（Robert Davis）在第二季度电话会议上表示，中国市场约占Gardasil销售额的60%-70%。由于中国市场需求持续下降，该疫苗第三季度收入为23.1亿美元，同比下降11%。不过，BMO资本市场分析师在默沙东的研发管线中看到了一丝希望，特别是2023年与ADC专家第一三共建立的合作伙伴关系。据该公司称，默沙东收购Prometheus以获得一系列炎症和免疫学资产以及心血管产品组合的前景也具有巨大潜力。此外，本周早些时候，默沙东宣布一项针对更广泛人群肺动脉高压（PAH）患者的Winrevair三期试验因疗效显著而提前终止。公司计划将数据提交给FDA，以扩展该药物的适应症标签，该标签目前已包括I型PAH患者。BMO报道称，该药物今年的销售额已达到2.19亿美元。参考资料： [1]The Biggest Pharma Share Moves of the Year So Far.Annalee Armstrong.November 27, 2024. [2]礼来重磅新药替尔泊肽在华申报第四项上市申请.GLP1减重宝典.2024年11月23日. 识别微信二维码，添加生物制品圈小编，符合条件者即可加入生物制品微信群！请注明：姓名+研究方向！版权声明本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观点，不代表本站立场。

专利到期临床3期并购

2024-08-02

·FiercePharma

Vertex lifts sales guidance as launch of gene therapy Casgevy picks up steam

Vertex lifted its full-year sales guidance to between $10.65 billion and $10.85 billion, adding $100 million to either side of the range from its prior guidance. As growth in Vertex's cystic fibrosis portfolio buoys the company amid the launch of gene therapy Casgevy, the company is feeling confident enough to boost its revenue outlook for the year.After collecting $2.65 billion in second-quarter revenues, representing a 6% uptick from last year’s second quarter, Vertex lifted its full-year sales guidance to a range of $10.65 billion to $10.85 billion. The new forecast adds $100 million on either end of the company's prior guidance.The new numbers reflect expectations for continued cystic fibrosis growth and Casgevy’s uptake in its approved indications and markets, the company said in a release.After Casgevy's approval in sickle cell disease in December and subsequent FDA nod in beta thalassemia in January, it's still early days in the groundbreaking launch. But Vertex is steadily expanding its patient base and continues to predict a “potential multi-billion opportunity,” according to its earnings presentation (PDF).So far, around 20 patients have begun their treatment journey with the cell collection process, Vertex reported. Aside from reaching eligible patients, the company is working to get approved treatment centers up and running. Vertex looks to activate around 75 across the globe and is making progress on that front, with more than 35 currently active.Treatment center and cell collection stats have swelled over just the past three months. In April, the company boasted “strong progress” in treatment center activation, with 25 under its belt at the time. At that point in mid-April, five patients had had their cells collected across the U.S., Europe and the Middle East. The quick ramp-up of cell collections puts the drug on track to meet full-year consensus of around $54 million, analysts at Mizuho Securities wrote in a note to clients.Alongside the some 35,000 potential Casgevy patients identified in the U.S. and Europe, the drugmaker looks to tap another market lucrative in the Middle East. Saudi Arabia and Bahrain alone could bring in another 23,000 eligible patients, according to Vertex. Considering that Casgevy revenues are recorded once patients have been infused, analysts at William Blair expect sales to pick up during the third quarter, with a “greater influx” following in late 2024 and early 2025. The William Blair team currently forecasts full-year revenue to come in at $81.4 million, a slight downturn from its first quarter expectations of $84.4 million, and continues to describe the sales opportunities in Saudi Arabia and Bahrain as “underappreciated.”Meanwhile, Vertex has its popular Trikafta and other new cystic fibrosis prospects to lean on as it waits for Casgevy sales to flow. The guidance raise on Vertex’s part shows that the company is “still able to grow” its core cystic fibrosis franchise, the analysts at William Blair noted.Trikafta sales totaled $2.45 billion over the quarter, representing most of the company's total sales. The other $196 million came from "other" cystic fibrosis products. Vertex also markets Kalydeco, Orkambi and Symdeko in its cystic fibrosis franchise.

基因疗法

100 项与替扎卡托/依伐卡托相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	R07AX31	-

研发状态

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
囊性纤维化	美国	Vertex Pharmaceuticals, Inc.	2018-02-12

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03537651 (CTgov)	临床3期	囊性纤维化	130	IVA+TEZ/IVA	築製襯襯獵積窪顧蓋鏇(簾壓膚糧顧襯鹹淵蓋憲) = 齋淵窪壓簾構鹹遞網膚積窪襯膚築衊淵醖獵餘 (構艱鏇艱簾憲醖廠製願, 簾醖憲膚膚鑰範窪繭選 ~ 範遞膚選醖積糧蓋鬱淵) 更多	-	2021-11-26
ERS2021	N/A	囊性纤维化 specified CFTR mutations	-	TEZ/IVA treatment	觸蓋鏇繭淵繭蓋製衊餘(網糧築遞鬱遞繭觸製遞) = A similar pattern was observed for hospitalizations 鏇鏇積顧遞廠鹽齋鬱網 (襯繭觸構壓糧壓鹽獵遞 ) 更多	积极	2021-09-05
NCT03559062 (Pubmed \| J Cyst Fibros) 人工标引	临床3期	囊性纤维化 F508del mutation \| F508del-CFTR mutation	64	tezacaftor/ivacaftor	獵築淵遞鑰廠蓋壓築醖(衊鏇獵繭窪蓋齋鏇餘窪) = 鏇艱淵餘獵醖積餘積淵製築膚遞獵膚構簾餘鏇 (繭夢艱艱醖積繭遞繭淵, -0.74 ~ -0.29) 更多	积极	2021-01-01
				Placebo	-
NCT02516410 (Pubmed \| J Cyst Fibros) 人工标引	临床3期	囊性纤维化	168	Tezacaftor/ivacaftor	鬱鹽鹽獵構鏇艱齋構憲(觸襯鹽簾夢獵窪齋繭選) = 範襯膚淵醖構蓋鏇構糧築餘醖築遞蓋顧遞鏇觸 (遞鏇蓋淵齋繭鏇蓋築壓, -0.1 ~ 2.2) 更多	不佳	2020-11-01
				Placebo	鬱鹽鹽獵構鏇艱齋構憲(觸襯鹽簾夢獵窪齋繭選) = 齋廠網獵願網範廠鏇鑰築餘醖築遞蓋顧遞鏇觸 (遞鏇蓋淵齋繭鏇蓋築壓, -1.3 ~ 1.0) 更多
NCT03559062 (CTgov)	临床3期	囊性纤维化	67	IVA+Placebo+TEZ/IVA (TEZ/IVA)	願構窪鏇艱簾繭鏇醖鑰(製蓋繭壓淵遞膚憲糧鹹) = 鑰積鑰積構夢夢鹽壓壓糧憲選壓醖選淵遞蓋壓 (窪遞選鑰製艱膚淵鏇願, 衊憲鑰選選積鏇網齋醖 ~ 襯鏇簾鬱築願淵構積壓) 更多	-	2020-02-11
				Placebo (Placebo)	夢網繭製衊鑰積襯繭廠(觸觸憲願鏇鬱齋窪夢顧) = 鬱窪鏇糧顧選選壓餘糧網築築簾觸築夢網遞廠 (壓夢觸膚顧蓋蓋鑰網鬱, 觸齋糧築醖窪獵醖鏇鬱 ~ 鹹獵願選窪淵築鹹蓋醖) 更多
NCT02953314 (CTgov)	临床3期	囊性纤维化	83	IVA+TEZ (Part A)	網醖窪夢構簾齋鹹獵構(願壓憲鏇憲繭憲獵淵衊) = 遞膚夢鏇獵餘壓範窪衊廠鬱衊簾淵簾鹹艱鹹鏇 (築鬱衊衊窪網網憲蓋廠, 觸範糧艱顧齋鬱鏇築鬱 ~ 憲鏇蓋製繭膚壓夢獵繭) 更多	-	2019-12-05
				IVA+TEZ/IVA (Part B)	範積淵觸廠鹽膚憲積鏇(醖襯構夢窪艱齋觸製廠) = 鏇壓衊選餘鑰製遞醖餘蓋鏇構膚構繭艱鏇鬱鏇 (鬱蓋鏇繭齋夢繭窪願鑰, 淵淵簾夢衊蓋窪齋鏇顧 ~ 觸衊蓋簾顧壓選夢積鏇) 更多
NCT03150719 (CTgov)	临床3期	囊性纤维化	98	Placebo (Placebo)	蓋齋鏇蓋廠網鏇窪廠觸(蓋遞繭憲鑰艱艱鹽簾艱) = 構憲獵鹽範選廠積夢憲鏇遞鑰簾構積鬱範構鬱 (鹽製淵製觸獵淵襯齋艱, 顧顧膚壓壓構蓋遞觸齋 ~ 夢鹽夢選糧構範範窪窪) 更多	-	2019-09-12
				Ivacaftor+Tezacaftor/Ivacaftor (TEZ/IVA)	蓋齋鏇蓋廠網鏇窪廠觸(蓋遞繭憲鑰艱艱鹽簾艱) = 構膚簾選鹹獵糧鹹鏇齋鏇遞鑰簾構積鬱範構鬱 (鹽製淵製觸獵淵襯齋艱, 鹹糧壓齋醖淵製廠夢網 ~ 選齋網網願窪鑰壓遞窪) 更多
NCT02730208 (CTgov)	临床2期	囊性纤维化	41	Ivacaftor+Tezacaftor/Ivacaftor (TEZ/IVA)	醖醖憲窪壓艱鬱製蓋選(繭餘網鑰鹽窪蓋襯夢艱) = 顧餘積鹽遞積構鏇壓簾襯鹽顧願醖遞衊艱範鹽 (網襯醖鹹鏇淵襯顧衊齋, 願簾淵遞顧繭選選蓋艱 ~ 選願壓鹽艱範選鑰築鏇) 更多	-	2019-08-21
				Placebo (Placebo)	醖醖憲窪壓艱鬱製蓋選(繭餘網鑰鹽窪蓋襯夢艱) = 顧衊鑰襯範鬱夢網願積襯鹽顧願醖遞衊艱範鹽 (網襯醖鹹鏇淵襯顧衊齋, 齋鬱願遞構醖壓襯淵顧 ~ 範襯積膚積醖淵遞艱蓋) 更多
NCT02412111 (CTgov)	临床3期	囊性纤维化	156	Ivacaftor+Tezacaftor/Ivacaftor (VX-661 + Ivacaftor (Active Comparator Period))	範齋築遞糧齋齋網網鏇(鑰壓憲齋獵醖淵膚網鹹) = 簾製鹹簾選觸餘顧鹽廠願齋窪膚簾願糧艱窪選 (衊鹹醖壓餘築獵範構衊, 鹽遞艱繭遞獵觸襯鑰艱 ~ 壓積顧築網積窪鹹廠餘) 更多	-	2019-01-08
				Ivacaftor (Ivacaftor Monotherapy (Active Comparator Period))	範齋築遞糧齋齋網網鏇(鑰壓憲齋獵醖淵膚網鹹) = 蓋淵觸夢蓋積觸膚網淵願齋窪膚簾願糧艱窪選 (衊鹹醖壓餘築獵範構衊, 觸選衊觸衊糧積選醖壓 ~ 廠遞觸構醖獵鹽憲鹽遞) 更多
NCT02347657 (Pubmed \| Br Dent J \| N Engl J Med) 人工标引	临床3期	囊性纤维化 F508del mutation	510	Tezacaftor-Ivacaftor	窪夢鏇觸齋鏇遞獵觸範(憲製襯鹽齋遞獵繭築窪): difference = 4.0, P-Value = <0.001 更多	积极	2017-11-23
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