BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the conditional approval of CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT)..

波士顿--（商业新闻短讯）--Vertex Pharmaceuticals Incorporated（纳斯达克：VRTX）今天宣布，欧洲药品管理局（EMA）人类使用药品委员会（CHMP）对卡奇维的有条件批准持积极意见™（exagamglogene autotemcel[exa cel]），一种CRISPR/Cas9基因编辑疗法，用于治疗严重镰状细胞病（SCD）和输血依赖性β地中海贫血（TDT）。。

If approved, exa-cel would be the only genetic therapy for patients in the European Union who are 12 years of age and older with either severe SCD with recurrent vaso-occlusive crises (VOCs) or TDT, for whom hematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available.

如果获得批准，exa-cel将是欧盟12岁及以上患有复发性血管闭塞性危象（VOCs）或TDT的严重SCD患者的唯一遗传疗法，造血干细胞（HSC）移植是合适的，并且没有人类白细胞抗原匹配的相关HSC供体。

An approval decision by the European Commission is expected in February 2024..

预计欧盟委员会将于2024年2月作出批准决定。。

“This positive opinion is yet another important regulatory milestone underscoring the potentially transformative benefit of CASGEVY for eligible patients with sickle cell and transfusion-dependent beta thalassemia,” said Nia Tatsis, Ph.D., Executive Vice President and Chief Regulatory and Quality Officer at Vertex..

Vertex执行副总裁兼首席监管和质量官Nia Tatsis博士说：“这一积极的观点是另一个重要的监管里程碑，强调了卡西维对符合条件的镰状细胞和输血依赖性β地中海贫血患者的潜在变革益处。”。。

“There is an urgent need for new potentially curative treatments in beta thalassemia and sickle cell disease, as people with these diseases still have a shorter life expectancy than the general population and an impaired quality of life,” said Franco Locatelli, M.D., Ph.D., Principal investigator in the CLIMB-111 and CLIMB-121 studies, Professor of Pediatrics at the Catholic University of the Sacred Heart, Rome, and Director of the Department of Pediatric Hematology and Oncology at the Bambino Gesù Children’s Hospital.

GRIMB-111和GRIMB-121研究的首席研究员、医学博士Franco Locatelli说：“迫切需要新的潜在治疗β地中海贫血和镰状细胞病的方法，因为患有这些疾病的人的预期寿命仍然比普通人群短，生活质量受损。”，罗马天主教圣心大学儿科教授，Bambino Gesù儿童医院儿科血液学和肿瘤学系主任。

“As an investigator, I have witnessed first-hand the transformative impact exa-cel can have on patients’ lives and I eagerly await the approval in the European Union.”.

“作为一名研究人员，我亲眼目睹了exa cel对患者生活产生的变革性影响，我急切地等待欧盟的批准。”。

About Sickle Cell Disease (SCD)

关于镰状细胞病（SCD）

SCD is a debilitating, progressive, life shortening genetic disease. SCD patients report health-related quality of life scores well below the general population and significant health care resource utilization. SCD affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body.

SCD是一种使人衰弱，进行性，缩短寿命的遗传疾病。SCD患者报告的健康相关生活质量得分远低于一般人群和显着的医疗保健资源利用率。SCD影响红细胞，红细胞是将氧气输送到身体所有器官和组织所必需的。

SCD causes severe pain, organ damage and shortened life span due to misshapen or “sickled” red blood cells. The clinical hallmark of SCD is vaso-occlusive crises (VOCs), which are caused by blockages of blood vessels by sickled red blood cells and result in severe and debilitating pain that can happen anywhere in the body at any time.

由于畸形或“镰状”红细胞，SCD会导致严重的疼痛，器官损伤和寿命缩短。SCD的临床标志是血管闭塞性危象（VOCs），这是由镰状红细胞阻塞血管引起的，并导致严重且衰弱的疼痛，这种疼痛可能随时发生在身体的任何地方。

SCD requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity. In Europe, the mean age of death for patients living with SCD is around 40 years. Stem cell transplant from a matched donor is a curative option but is only available to a small fraction of people living with SCD because of the lack of available donors..

SCD需要终身治疗和大量使用医疗保健资源，最终导致预期寿命缩短，生活质量下降，终身收入和生产力下降。在欧洲，SCD患者的平均死亡年龄约为40岁。来自匹配捐赠者的干细胞移植是一种治疗选择，但由于缺乏可用的捐赠者，只有一小部分SCD患者可用。。

About Transfusion-Dependent Beta Thalassemia (TDT)

关于输血依赖性β地中海贫血（TDT）

TDT is a serious, life-threatening genetic disease. TDT patients report health-related quality of life scores below the general population and significant health care resource utilization. TDT requires frequent blood transfusions and iron chelation therapy throughout a person’s life. Due to anemia, patients living with TDT may experience fatigue and shortness of breath, and infants may develop failure to thrive, jaundice and feeding problems.

TDT是一种严重的威胁生命的遗传疾病。TDT患者报告的健康相关生活质量得分低于普通人群，并且医疗保健资源利用率很高。TDT需要在一个人的一生中频繁输血和铁螯合疗法。由于贫血，患有TDT的患者可能会出现疲劳和呼吸急促，婴儿可能会出现failure壮成长、黄疸和喂养问题。

Complications of TDT can also include an enlarged spleen, liver and/or heart, misshapen bones and delayed puberty. TDT requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity.

TDT的并发症还可能包括脾脏，肝脏和/或心脏肿大，骨骼畸形和青春期延迟。TDT需要终身治疗和大量使用医疗保健资源，最终导致预期寿命缩短，生活质量下降，终身收入和生产力下降。

In Europe, the mean age of death for patients living with TDT is 50-55 years. Stem cell transplant from a matched donor is a curative option but is only available to a small fraction of people living with TDT because of the lack of available donors..

在欧洲，患有TDT的患者的平均死亡年龄为50-55岁。来自匹配供体的干细胞移植是一种治疗选择，但由于缺乏可用的供体，只有一小部分患有TDT的人可用。。

About CASGEVY™ (exagamglogene autotemcel [exa-cel])

关于CASGEVY™（exagaglogene autoemcel）

CASGEVY™ is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells.

卡西维™是一种非病毒，离体CRISPR/Cas9基因编辑的细胞疗法，适用于符合条件的SCD或TDT患者，其中患者自身的造血干细胞和祖细胞通过精确的双链断裂在BCL11A基因的红系特异性增强子区域进行编辑。这种编辑导致红细胞中产生高水平的胎儿血红蛋白（HbF；血红蛋白F）。

HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and alleviate transfusion requirements for patients with TDT..

HbF是胎儿发育过程中天然存在的含氧血红蛋白的形式，出生后会转变为成人形式的血红蛋白。CASGEVY已被证明可以减少或消除SCD患者的挥发性有机化合物，并减轻TDT患者的输血需求。。

The use of CASGEVY in the European Union remains investigational.

卡西维在欧盟的使用仍在调查中。

CASGEVY is approved in the U.S. to treat people aged 12 years and older with SCD who have recurrent VOCs. CASGEVY was granted a conditional marketing authorization in Great Britain by the U.K. Medicines and Healthcare products Regulatory Agency for patients 12 years of age and older with SCD characterized by recurrent VOCs or TDT, for whom hematopoietic stem cell transplantation is appropriate and a human leukocyte antigen matched related hematopoietic stem cell donor is not available.

CASGEVY在美国被批准用于治疗12岁及以上患有复发性挥发性有机化合物的SCD患者。英国药品和保健品管理局（U.K.Medicines and Healthcare products Regulatory Agency）为12岁及以上以复发性VOCs或TDT为特征的SCD患者授予了英国有条件的上市许可，这些患者适合进行造血干细胞移植，并且没有人类白细胞抗原匹配的相关造血干细胞供体。

CASGEVY is currently under review by the Saudi Food and Drug Authority for both SCD and TDT and the U.S. Food and Drug Administration for TDT..

卡西维目前正在接受沙特食品和药物管理局对SCD和TDT的审查，以及美国食品和药物管理局对TDT的审查。。

About Conditional Marketing Authorizations (CMAs)

关于有条件营销授权（CMA）

CMAs are for medicines that fulfil a significant unmet medical need such as being for serious and life-threatening diseases, where no satisfactory treatment methods are available or where the medicine offers a major therapeutic advantage. A CMA is granted where comprehensive clinical data is not yet complete, but the benefit of the medicine to address a significant unmet need outweighs the need for data that will become available in the future.

CMA用于满足严重未满足的医疗需求的药物，例如用于严重和危及生命的疾病，这些疾病没有令人满意的治疗方法，或者药物具有主要的治疗优势。如果综合临床数据尚未完成，则授予CMA，但该药物解决重大未满足需求的益处超过了对未来可用数据的需求。

CMAs are valid for one year and renewable annually with ongoing regulatory review of data..

CMA有效期为一年，每年可续展，并对数据进行持续的监管审查。。

U.S. INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR CASGEVY (exagamglogene autotemcel)

U、 美国CASGEVY（exagamglogene autotemcel）的适应症和重要安全信息

WHAT IS CASGEVY?

什么是卡西维？

CASGEVY is a one-time therapy used to treat people aged 12 years and older with sickle cell disease (SCD) who have frequent vaso-occlusive crises or VOCs.

CASGEVY是一种一次性疗法，用于治疗12岁及以上镰状细胞病（SCD）患者，这些患者经常出现血管闭塞性危机或VOC。

CASGEVY is made specifically for each patient, using the patient’s own edited blood stem cells, and increases the production of a special type of hemoglobin called hemoglobin F (fetal hemoglobin or HbF). Having more HbF increases overall hemoglobin levels and has been shown to improve the production and function of red blood cells.

CASGEVY专门为每位患者制造，使用患者自己编辑的血液干细胞，并增加一种称为血红蛋白F（胎儿血红蛋白或HbF）的特殊类型血红蛋白的产生。拥有更多的HbF会增加整体血红蛋白水平，并已被证明可以改善红细胞的产生和功能。

This can eliminate VOCs in people with SCD..

这可以消除SCD患者体内的挥发性有机化合物。。

IMPORTANT SAFETY INFORMATION

重要安全信息

What is the most important information I should know about CASGEVY?

关于卡西维，我应该知道的最重要的信息是什么？

After treatment with CASGEVY, you will have fewer blood cells for a while until CASGEVY takes hold (engrafts) into your bone marrow. This includes low levels of platelets (cells that usually help the blood to clot) and white blood cells (cells that usually fight infections). Your doctor will monitor this and give you treatment as required.

在用卡西维治疗后，你的血细胞会减少一段时间，直到卡西维（植入）你的骨髓。这包括低水平的血小板（通常帮助血液凝结的细胞）和白细胞（通常抵抗感染的细胞）。您的医生将对此进行监测，并根据需要为您提供治疗。

The doctor will tell you when blood cell levels return to safe levels..

当血细胞水平恢复到安全水平时，医生会告诉你。。

Tell your healthcare provider right away if you experience any of the following, which could be signs of low levels of platelet cells:

如果您遇到以下任何情况，请立即告诉您的医疗保健提供者，这可能是血小板水平低的迹象：

severe headache

严重头痛

abnormal bruising

异常瘀伤

prolonged bleeding

长时间出血

bleeding without injury such as nosebleeds; bleeding from gums; blood in your urine, stool, or vomit; or coughing up blood

出血没有受伤，如流鼻血；牙龈出血；尿液、粪便或呕吐物中有血液；或者咳血

Tell your healthcare provider right away if you experience any of the following, which could be signs of low levels of white blood cells:

如果您遇到以下任何情况，请立即告诉您的医疗保健提供者，这可能是白细胞水平低的迹象：

fever

发烧

chills

寒战

infections

感染

You may experience side effects associated with other medicines administered as part of the treatment regimen with CASGEVY. Talk to your physician regarding those possible side effects. Your healthcare provider may give you other medicines to treat your side effects.

您可能会遇到与其他药物相关的副作用，这些药物是CASGEVY治疗方案的一部分。与你的医生讨论这些可能的副作用。您的医疗保健提供者可能会给您其他药物来治疗您的副作用。

How will I receive CASGEVY?

我将如何接收卡西维？

Your healthcare provider will give you other medicines, including a conditioning medicine, as part of your treatment with CASGEVY. It's important to talk to your healthcare provider about the risks and benefits of all medicines involved in your treatment.

您的医疗保健提供者将为您提供其他药物，包括调理药物，作为您使用卡西维治疗的一部分。与您的医疗保健提供者谈论您治疗中涉及的所有药物的风险和益处非常重要。

After receiving the conditioning medicine, it may not be possible for you to become pregnant or father a child. You should discuss options for fertility preservation with your healthcare provider before treatment.

服用调理药后，您可能不可能怀孕或生孩子。治疗前，你应该与你的医疗保健提供者讨论保留生育能力的选择。

STEP 1: Before CASGEVY treatment, a doctor will give you a mobilization medicine. This medicine moves blood stem cells from your bone marrow into the blood stream. The blood stem cells are then collected in a machine that separates the different blood cells (this is called apheresis). This entire process may happen more than once.

第一步：在卡西维治疗之前，医生会给你一种动员药。这种药物将血液干细胞从你的骨髓转移到血液中。然后将血液干细胞收集到一台分离不同血细胞的机器中（这称为单采）。整个过程可能不止一次发生。

Each time, it can take up to one week..

每次都可能需要一周的时间。。

During this step, rescue cells are also collected and stored at the hospital. These are your existing blood stem cells and are kept untreated just in case there is a problem in the treatment process. If CASGEVY cannot be given after the conditioning medicine, or if the modified blood stem cells do not take hold (engraft) in the body, these rescue cells will be given back to you.

在此步骤中，救援细胞也被收集并储存在医院。这些是你现有的血液干细胞，未经处理，以防治疗过程中出现问题。如果在服用调理药后不能给予卡奇维，或者如果经过修饰的血液干细胞不能在体内存活（植入），这些救援细胞将被归还给你。

If you are given rescue cells, you will not have any treatment benefit from CASGEVY..

如果你得到救援细胞，你将不会从卡西维那里获得任何治疗益处。。

STEP 2: After they are collected, your blood stem cells will be sent to the manufacturing site where they are used to make CASGEVY. It may take up to 6 months from the time your cells are collected to manufacture and test CASGEVY before it is sent back to your healthcare provider.

第二步：采集后，你的血液干细胞将被送到生产基地，用于制造卡奇维。从收集细胞制造和测试CASGEVY到将其送回医疗保健提供者，可能需要长达6个月的时间。

STEP 3: Shortly before your stem cell transplant, your healthcare provider will give you a conditioning medicine for a few days in hospital. This will prepare you for treatment by clearing cells from the bone marrow, so they can be replaced with the modified cells in CASGEVY. After you are given this medicine, your blood cell levels will fall to very low levels.

第三步：在你的干细胞移植前不久，你的医疗保健提供者会给你一种调理药物，让你在医院呆几天。这将通过清除骨髓中的细胞为您的治疗做好准备，因此可以用CASGEVY中的修饰细胞替代它们。服用这种药后，你的血细胞水平会降至非常低的水平。

You will stay in the hospital for this step and remain in the hospital until after the infusion with CASGEVY..

这一步你将留在医院，并一直留在医院，直到输注了卡西维。。

STEP 4: One or more vials of CASGEVY will be given into a vein (intravenous infusion) over a short period of time.

步骤4：在短时间内将一瓶或多瓶CASGEVY注入静脉（静脉输注）。

After the CASGEVY infusion, you will stay in hospital so that your healthcare provider can closely monitor your recovery. This can take 4-6 weeks, but times can vary. Your healthcare provider will decide when you can go home.

卡奇维输液后，您将留在医院，以便您的医疗保健提供者可以密切监测您的康复情况。这可能需要4-6周，但时间可能会有所不同。您的医疗保健提供者将决定您何时可以回家。

What should I avoid after receiving CASGEVY?

收到卡奇维后我应该避免什么？

Do not donate blood, organs, tissues, or cells at any time in the future

今后任何时候都不要捐献血液、器官、组织或细胞

What are the possible or reasonably likely side effects of CASGEVY?

CASGEVY可能或合理可能的副作用是什么？

The most common side effects of CASGEVY include:

卡西维最常见的副作用包括：

Low levels of platelet cells, which may reduce the ability of blood to clot and may cause bleeding

血小板水平低，这可能会降低血液凝结的能力，并可能导致出血

Low levels of white blood cells, which may make you more susceptible to infection

白细胞水平低，这可能使你更容易感染

Your healthcare provider will test your blood to check for low levels of blood cells (including platelets and white blood cells). Tell your healthcare provider right away if you get any of the following symptoms:

您的医疗保健提供者将测试您的血液，以检查低水平的血细胞（包括血小板和白细胞）。如果您出现以下任何症状，请立即告知您的医疗保健提供者：

fever

发烧

chills

寒战

infections

感染

severe headache

严重头痛

abnormal bruising

异常瘀伤

prolonged bleeding

长时间出血

bleeding without injury such as nosebleeds; bleeding from gums; blood in your urine, stool, or vomit; or coughing up blood

出血没有受伤，如流鼻血；牙龈出血；尿液、粪便或呕吐物中有血液；或者咳血

These are not all the possible side effects of CASGEVY. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

这些并不是卡西维所有可能的副作用。打电话给你的医生，询问有关副作用的医疗建议。您可以通过1-800-FDA-1088向FDA报告副作用。

General information about the safe and effective use of CASGEVY

关于安全有效使用卡西维的一般信息

Talk to your healthcare provider about any health concerns.

与您的医疗保健提供者谈论任何健康问题。

Please see full Prescribing Information including Patient Information for CASGEVY.

请参阅完整的处方信息，包括CASGEVY的患者信息。

About Vertex

关于顶点

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying cause of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases.

Vertex是一家全球生物技术公司，投资于科学创新，为患有严重疾病的人创造变革性药物。该公司已批准用于治疗多种慢性，缩短寿命的遗传疾病（囊性纤维化，镰状细胞病和输血依赖性β地中海贫血）的根本原因的药物，并继续推进这些疾病的临床和研究计划。

Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including APOL1-mediated kidney disease, acute and neuropathic pain, type 1 diabetes and alpha-1 antitrypsin deficiency..

Vertex还在其他严重疾病的一系列模式中拥有强大的临床研究治疗渠道，对因果人类生物学有深入了解，包括APOL1介导的肾脏疾病，急性和神经性疼痛，1型糖尿病和α-1抗胰蛋白酶缺乏症。。

Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 14 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For.

Vertex成立于1989年，全球总部位于波士顿，国际总部位于伦敦。此外，该公司在北美、欧洲、澳大利亚和拉丁美洲设有研发基地和商业办事处。Vertex一直被公认为行业最佳工作地点之一，包括连续14年入选《科学》杂志的最佳雇主名单，以及《财富》杂志的100家最佳工作公司之一。

For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on LinkedIn, Facebook, Instagram, YouTube and Twitter/X..

有关公司更新和Vertex创新历史的更多信息，请访问www.vrtx.com或在LinkedIn、Facebook、Instagram、YouTube和Twitter/X上关注我们。。

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Vertex Special Note Regarding Forward-Looking Statements

Vertex关于前瞻性陈述的特别说明

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements made by Nia Tatsis, Ph.D., and Franco Locatelli, M.D., Ph.D., in this press release and statements regarding our expectations for regulatory approval for CASGEVY™, including anticipated timing of approval, the anticipated population eligible for treatment, our expectation that, if approved, CASGEVY will be the only genetic therapy available for eligible patients in the European Union, and our beliefs regarding the benefits of our genetic therapy.

本新闻稿包含1995年《私人证券诉讼改革法案》修订版中定义的前瞻性声明，包括但不限于Nia Tatsis博士和Franco Locatelli博士在本新闻稿中的声明，以及关于我们对卡西维监管批准的期望的声明™,包括预期的批准时间，预期的符合治疗条件的人群，我们的期望，如果获得批准，卡西维将是欧盟符合条件的患者唯一可用的基因疗法，以及我们对基因疗法益处的信念。

While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements.

虽然Vertex认为本新闻稿中包含的前瞻性陈述是准确的，但这些前瞻性陈述仅代表截至本新闻稿发布之日的公司信念，并且存在许多风险和不确定性，可能导致实际事件或结果与此类前瞻性陈述所表达或暗示的事件或结果存在重大差异。

Those risks and uncertainties include, among other things, that regulatory authorities may not approve CASGEVY on a timely basis or at all, and other risks listed under the heading “Risk Factors” in Vertex's annual report and in subsequent filings filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com and www.sec.gov.

这些风险和不确定性包括监管机构可能不会及时或根本不批准卡西维，以及Vertex年报和随后向证券交易委员会提交的文件中“风险因素”标题下列出的其他风险，这些风险和不确定性可通过公司网站www.vrtx.com和www.sec.gov获得。

You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available..

你不应该过分依赖这些陈述。Vertex不承担在新信息可用时更新本新闻稿中包含的信息的任何义务。。