CLINICAL TRIAL: MYOZYME? TEMPORARY ACCESS PROGRAM (MTAP)

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 Myozyme临时访问计划的目标是让美国庞贝病成年患者在有限的时间内获得由扩大生产工艺生产的Myozyme，直到这种规模的生产获得美国食品和药物管理局(FDA)的商业用途批准。 Myozyme自2003年初以来一直用于治疗庞贝病患者的临床试验。2006年4月28日，美国市场批准使用Myozyme治疗庞贝病。Genzyme已经扩大并修改了现有的生产Myozyme的工艺，以便更大规模地生产Myozyme，目前该工艺已被批准在美国以外的地方用于商业用途，以继续向患者提供供应。这种规模的材料在美国还没有被证明可以用于商业用途，但它被用于正在进行的临床试验。根据这一准入计划，从扩大生产过程中生产的Myozyme将在有限的时间内进行管理，直到生产规模获得FDA的批准。

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 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.