Vertex Pharmaceuticals (VRTX) , along with CRISPR Therapeutics (CRSP) , announced that the FDA expanded the label of their one-shot gene therapy Casgevy to treat transfusion-dependent beta thalassemia (TDT) in patients aged 12 years and older. The approval comes just a month after the FDA approved the therapy for sickle cell disease (SCD) in the same age group. Following this initial approval, Casgevy became the first gene therapy utilizing the Nobel prize- winning CRISPR technology. This technology can sel ...

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BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa- cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older. “ On the heels of the historic FDA approval of CASGEVY for sickle cell disease, it is exciting to now secure approval for TDT well ahead of the PDUFA date,” said Reshma Kew ...

TROY, Mich., Jan. 10, 2024 /PRNewswire/ -- The Editorial Advisory and Securities Review Committee of BetterInvesting Magazine today announced Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) as its "Stock to Study" and Martin Marietta Materials Inc. (NYSE: MLM) as its "Undervalued Stock" in the March 2024 issue for investors' informational and educational use. "The S&P500 Index notched a total return gain of more than 26% in 2023, although a small number of individual stocks had an outsize influence over the inde ...

股价表现 - Vertex Pharmaceuticals (VRTX) 最近的股价为 $421.01，较前一个交易日收盘价上涨了 +0.45% [1] - 过去一个月，该公司股价上涨了 17.64%，超过了医疗行业的 6.58% 和标普500指数的 3.5% [2] 盈利预期 - 投资者将密切关注 Vertex Pharmaceuticals 的盈利表现，预计公司将报告每股收益为 $4.08，较去年同期增长了 8.51% [3] 分析师评级 - 分析师对 Vertex Pharmaceuticals 的估值也有所变化，正面的估值修订表明分析师对公司业绩和利润潜力的信心 [4] 行业排名 - 医疗 - 生物医学和遗传学行业目前的 Zacks Industry Rank 为 66，在所有行业中排名前 27% [9] - Zacks Industry Rank 衡量了各行业群体的实力，研究显示排名前 50% 的行业胜过后 50% 的行业 [10]

BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the Saudi Food and Drug Authority (SFDA) granted Marketing Authorization for CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). CASGEVY is approved for the treatment of people 12 years of age and older with SCD or TDT. The Kingdom of Saudi Arabia has among the highest prevalence rates o ...

业绩总结 - Vertex Pharmaceuticals Incorporated 在 2023 年的收入指导为 98.5 亿美元，实现了两位数增长[38] - Vertex 在 2024 年将推出 CASGEVY，开始多元化收入[40] - Vertex 在研发领域取得了快速进展，继续在内部和外部创新方面投资[37] 新产品和新技术研发 - Vertex 的研发策略交付了成果，已经在 5 个疾病领域推出产品[39] - Vertex 在疼痛领域持续进行创新和领导[23] - Vertex 在糖尿病领域有三个项目，其中 VX-880 的试验显示出前所未有的结果[33] 市场扩张和并购 - Vertex 在 2023 年进行了约 10 笔交易，以补充、加强或加速公司在疾病领域的内部努力[38] - 美国急性疼痛患者数量为8000万，市场规模达数十亿美元[43] - Vertex已与Synergy达成协议，覆盖1亿商业人群[53] 未来展望 - CASGEVY预计将成为多十亿美元的机会[50] - CASGEVY目标是与囊性纤维化一样，治疗众多患者，但治疗过程较长[52] - Vertex为CASGEVY患者提供旅行和住宿支持[57]

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) J.P. Morgan 42nd Annual Healthcare Conference January 8, 2024 11:15 AM ET Company Participants Jessica Fye - J.P. Morgan Conference Call Participants Reshma Kewalramani - Chief Executive Officer and President Jessica Fye Good morning, everyone. We're going to get started. My name is Jess Fye, I'm a Senior Biotech Analyst at J.P. Morgan, continuing the 42nd Annual Healthcare Conference this morning with Vertex. I'm joined by the Company's CEO, Reshma Kewalram ...

Vertex Pharmaceuticals (VRTX) 盈利情况 - Vertex 上个季度每股盈利为 $4.08，超过 Zacks 预期的 $3.92，意味着盈利超出预期 4.08%[3] - 最新的预测显示 Vertex 的盈利预期在上升，Zacks Earnings ESP 为正值，这是一个盈利超出预期的好迹象[4] - 公司研究显示，具有正面 Earnings ESP 和 Zacks Rank 3 (Hold) 或更好的股票，有将近 70% 的概率会产生正面的盈利惊喜[5] Vertex Pharmaceuticals (VRTX) 盈利预期 - Zacks Earnings ESP 比较了最准确的预测与 Zacks 预期之间的差异，这个指标结合 Zacks Rank 3 (Hold) 表明 Vertex 可能会再次超出预期[6] - Vertex 目前的 Earnings ESP 为 +0.36%，分析师对公司的盈利前景最近变得乐观，这一正面的 Earnings ESP 结合 Zacks Rank 3 (Hold) 表明可能会再次超出预期[7] - 负面的 Earnings ESP 不代表盈利未达预期，但负值会降低这一指标的预测能力[8] Vertex Pharmaceuticals (VRTX) 盈利策略 - 公司强调，许多公司最终会超过共识的每股盈利预期，但这并不一定是他们的股价上涨的唯一基础[9] - 重要的是在季度发布前检查公司的 Earnings ESP，以增加成功的几率[10]

Vertex Pharmaceuticals Incorporated业务发展 - Vertex Pharmaceuticals Incorporated宣布2023年在囊性纤维化业务表现强劲，加速发展阶段管线，为2024年带来巨大动力[1] - Vertex已获得CASGEVY在美国、英国和巴林的批准，以及欧洲药品管理局对CASGEVY用于SCD和TDT的正面意见，预计CASGEVY在TDT方面的PDUFA行动日期为2024年3月30日[2] 新药研发进展 - Vertex完成了三项评估下一代三联组合CFTR调节剂疗法vanzacaftor/tezacaftor/deutivacaftor的关键研究，预计将在2024年初公布结果[2] - Vertex已完成VX-548用于中度至重度急性疼痛的三项3期研究，预计将在2024年初公布所有三项研究的结果[3] - Vertex计划在2024年启动VX-993的口服制剂的2期研究，用于治疗中度至重度急性疼痛[3] - Vertex计划在2024年启动VX-993的口服制剂用于治疗PNP的2期研究[5] 临床试验和研究计划 - Vertex已完成对Inaxaplin用于治疗AMKD患者的2B剂量范围部分的招募，并预计将在2024年第一季度选择Inaxaplin剂量并开始2/3关键试验的3期[6] - Vertex已完成VX-880用于T1D患者的1/2期研究的A、B和C部分的招募，并展示了良好的疗效[7] - VX-880的安全性与免疫抑制剂、围手术期和过往病史一致，但已发生两例与VX-880无关的患者死亡，公司已将研究暂停[9] 未来发展计划 - Vertex计划在2024年与监管机构会面，预计将VX-548推进至关键发展阶段，以治疗周围神经病性疼痛[4] - Vertex计划在2024年与FDA合作进行VX-670的研究，并在英国启动VX-670的临床试验[12] - Vertex已完成VX-407用于治疗ADPKD的IND前研究，并计划在2024年在健康志愿者中开始1期临床试验[14]