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ROCTAVIAN™(valoctocogene roxaparvovec-rvox) gene therapy

ROCTAVIAN is a gene therapy indicated for the treatment of adults with severe hemophilia A (congenital Factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) as detected by an 皇冠体育官网-approved test. The one-time, 单剂量输注是为了取代突变基因的功能, allowing people with severe hemophilia A to produce their own FVIII to prevent or reduce the occurrence of bleeding.

You should not take ROCTAVIAN if you have an active infection or if you have a long-term infection that is not controlled by the medicines you take 肝脏有疤痕(明显的肝纤维化或肝硬化) or 对甘露醇过敏(ROCTAVIAN中的非活性成分). 请参阅以下重要安全资讯, 完整的处方信息, and Patient Information.


适应症和重要安全信息

What is ROCTAVIAN?
ROCTAVIAN is a one-time gene therapy used for the treatment of adults with severe hemophilia A who do not have antibodies to the virus, AAV5是由血液测试确定的.

ROCTAVIAN使用了一种改良病毒, called a vector, to deliver a working copy of the factor VIII gene to liver cells to enable your body to produce clotting factor on its own, which helps the blood to clot and prevents or reduces the occurrence of bleeding. The modified virus does not contain viral DNA and does not cause disease in humans.

不要服用ROCTAVIAN,如果你:

  • have an active infection or if you have a long-term infection that is not controlled by the medicines you take
  • 肝脏有疤痕(明显的肝纤维化或肝硬化)
  • 对甘露醇过敏(ROCTAVIAN中的非活性成分)

关于ROCTAVIAN我应该知道的最重要的信息是什么?
ROCTAVIAN在输注期间和输注后可能会引起严重的副作用:

  • 在注射期间和注射后几小时内, tell your doctor or nurse immediately about any symptoms you experience including hives or other rashes, itching, sneezing, coughing, difficulty breathing, runny nose, watery eyes, tingling throat, nausea (feeling sick), diarrhea, low blood pressure, rapid heartbeat, 头晕(几近晕倒), fever, chills, and shivering. Talk to your doctor about what to do if you experience any side effects after you leave the infusion.
  • Before and regularly following administration of ROCTAVIAN your doctor will perform blood tests to check your liver health. Make sure you obtain these blood tests during the specified time your doctor instructs you to. 根据你的肝脏检查结果, you may need to take corticosteroids or another medicine for a period of time (several months or longer) to help decrease liver enzyme levels, 哪些药物在服用时可能会产生副作用. Talk to your doctor about these side effects and what you need to do to improve and maintain your liver’s health.
  • 有活性因子VIII抑制剂的患者不应服用ROCTAVIAN. Following administration your doctor will monitor you for inhibitors and you will have regular factor level testing. 如果服用ROCTAVIAN后开始出血,请咨询医生, 为了让你的医生评估是否需要额外的检查或治疗.
  • 取决于你的风险因素, an improvement in factor VIII levels may mean an increased possibility of unwanted blood clots (so called “thromboses”, 静脉或动脉). You and your doctor should discuss your risk factors before and after treatment and how to recognize symptoms of unwanted clots and what to do if you think you may have one.
  • ROCTAVIAN可以将自己插入人体细胞的DNA中. 插入对这些细胞的影响尚不清楚, 但这些事件可能会导致理论上的癌症风险. 目前还没有ROCTAVIAN治疗引起癌症的病例报告. Your doctor may perform regular monitoring if you have pre-existing risk factors for developing liver cancer. In the event of cancer, your doctor may send a sample to 皇冠体育娱乐场网址 Pharmaceutical Inc. for further testing.

在我得到ROCTAVIAN之前,我应该告诉我的医生什么?
和你的医生谈谈以下几点:

  • 您的健康状况包括:
    • any general risk factors for unwanted blood clots and for cardiovascular disease
    • 如果你的免疫系统抵抗感染的能力下降了
    • 如果你有抑制剂或者有因子VIII抑制剂的历史
  • 你正在服用的所有药物或你计划服用的新药, including prescription and nonprescription drugs, vitamins, herbal supplements, and vaccines
  • If you have a female partner that plans to become pregnant within 6 months of treatment

服用ROCTAVIAN后应该避免什么?

  • 第一年避免饮酒. Talk to your doctor about how much alcohol may be acceptable after the first year.
  • 你和任何女性伴侣必须在6个月内避免怀孕. 与你的医生讨论哪种避孕方法是合适的.
  • 治疗后至少6个月内不要捐献精液.
  • 不捐献血液、器官、组织或细胞.

ROCTAVIAN可能有哪些副作用?

  • ROCTAVIAN最常见的副作用是:
    • Nausea, fatigue, headache, infusion-related reactions, vomiting, and abdominal pain.
    • Changes to laboratory results from blood tests that measure your liver health and other ways your body is responding to ROCTAVIAN.

在得到ROCTAVIAN之前,我还需要知道哪些信息?

  • 未来再次接受基因治疗: It is not yet known whether or under which conditions you may be able to repeat ROCTAVIAN treatment or use another gene therapy.
  • 血友病治疗登记: ROCTAVIAN治疗后, you will be asked to enroll in a 15 year registry to help study the long term safety of the treatment and how well it continues to work.
  • 了解ROCTAVIAN的风险和益处: 虽然大多数患者从ROCTAVIAN中获益, 治疗效果和持续时间可能有所不同. 有些患者没有从ROCTAVIAN中获益. 不可能预测患者是否会受益以及受益程度. After administration, your doctor will monitor your lab tests and talk to you about whether you can stop prophylaxis, 你是否应该再次开始预防, 以及你是否应该以及如何治疗任何手术, procedures, injuries, or bleeds.和你的医生谈谈ROCTAVIAN的潜在风险和益处. 病人是否从中受益, 这里和你的医生讨论的风险仍然适用.

这些并不是ROCTAVIAN可能产生的所有副作用. 向你的医生咨询有关副作用的医疗建议. 你可以向皇冠体育娱乐场网址制药公司报告副作用. 1-866-906-6100或皇冠体育官网 1-800-皇冠体育官网-1088.

请参阅附带的其他安全信息 Prescribing Information and Patient Information.


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