CLINICAL TRIAL: MYOZYME? TEMPORARY ACCESS PROGRAM (MTAP)

临床试验：肌酶？

• 批准号: 7951008
• 负责人: BRUCE A BARSHOP
• 金额: $ 1.7万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN DIEGO
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-12-01 至 2009-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7951008
• 关键词: Adult; Clinical Research; Clinical Trials; Computer Retrieval of Information on Scientific Projects Database; Funding; Glycogen storage disease type II; Grant; Institution; Marketing; Patients; Production; Research; Research Personnel; Resources; Source; Time; United States Food and Drug Administration; United States National Institutes of Health; manufacturing process; programs; scale up

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The objective of the Myozyme Temporary Access Program is to provide adult patients with Pompe disease in the US access to Myozyme produced from a scaled up manufacturing process for a limited time until production at this scale is approved for commercial use by the Food and Drug Administration (FDA). Myozyme has been used since early 2003 in clinical trials to treat patients with Pompe disease. Market approval in the US for the use of Myozyme to treat Pompe disease was granted on 4/28/06. Genzyme has scaled up and modified the existing manufacturing process for production of Myozyme at a larger scale, which is currently approved for commercial use outside of the US, in order to provide a continued supply to patients. This scale of material is not yet pproved for commercial use in the US; however it is used in ongoing clinical trials. Myozyme, produced from the scaled-up manufacturing process will be administered for a limited time under this access program until the manufacturing scale is approved by the FDA.

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 美而赞临时使用计划的目的是为美国的庞贝氏症成人患者提供美而赞的有限使用权，美而赞是在美国食品药品监督管理局（FDA）批准该规模的生产用于商业用途之前，通过扩大生产工艺生产的。 自2003年初以来，美而赞一直用于临床试验，治疗庞贝氏症患者。美而赞用于治疗庞贝氏症的美国上市批准于2006年4月28日获得。Genzyme已经扩大并修改了现有的生产工艺，以更大规模生产美而赞，目前已批准在美国以外地区商业使用，以便为患者提供持续供应。这种规模的材料尚未在美国获得商业用途的批准;但它已用于正在进行的临床试验。在FDA批准生产规模之前，将根据本准入计划在有限的时间内给予采用规模扩大生产工艺生产的美而赞。