CLINICAL TRIAL: ALGLUCOSIDASE ALFA TEMPORARY ACCESS PROGRAM (ATAP) PROTOCOL AGLU

该子项目是利用 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来源获得主要资金， 因此可以在其他CRISP条目中表示。列出的机构是 中心，不一定是研究者的机构。 美而赞临时使用计划的目的是在有限的时间内为美国的庞贝氏症成人患者提供美而赞，该美而赞是通过扩大生产工艺生产的，直到该规模的生产完成。 经美国食品药品监督管理局（FDA）批准用于商业用途。 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 现有的生产工艺用于大规模生产美而赞，目前已在美国以外批准用于商业用途，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。根据本准入计划，将在有限时间内对规模扩大生产工艺生产的美而赞进行给药，直至 生产规模已获FDA批准。

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 28 April 2006. 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.