MYOZYME TEMPORARY ACCESS PROGRAM (MTAP)

MYOZYME 临时访问计划 (MTAP)

• 批准号: 8167320
• 负责人: Kenneth Rosenbaum
• 金额: $ 1.13万
• 依托单位: CHILDREN'S RESEARCH INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-01-20 至 2010-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8167320
• 关键词: Adolescent; Adult; Clinical Research; Clinical Trials; Computer Retrieval of Information on Scientific Projects Database; Disease; Dose; Ensure; FDA approved; Funding; Glycogen storage disease type II; Grant; Institution; Intravenous; Licensing; Licensure; Patients; Process; Production; Protocols documentation; Recombinant Proteins; Research; Research Personnel; Resources; Site; Source; Time; United States National Institutes of Health; enzyme replacement therapy; infancy; 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. Myozyme Temporary Access Program (MTAP) is to provide patients with Pompe disease in the US access to intravenous enzyme replacement therapy (ERT) with Myozyme produced from a scaled-up manufacturing process for a limited time until production at this scale is approved for commercial use by the FDA. Myozyme produced via this scaled-up process has been used in numerous clinical studies to date, including the ongoing clinical trial in patients with late-onset Pompe disease (AGLU02704) (CNMC is one site for this trial) and by all commercial patients outside the US. As is often the case with recombinant protein manufacture, the process was developed at a smaller volume, optimized, and subsequently scaled up to produce larger volumes. Genzyme is pursuing licensure of product produced from the scaled-up manufacturing process via a supplement to the existing Biologics License Application for Myozyme. This protocol, to allow adult patients who require larger doses of Myozyme to receive this large scale manufactured product, will also ensure an adequate supply of FDA-approved smaller volume produced Myozyme to be used for infantile and juvenile patients with Pomoe disease.

该副本是利用众多研究子项目之一 由NIH/NCRR资助的中心赠款提供的资源。子弹和 调查员（PI）可能已经从其他NIH来源获得了主要资金， 因此可以在其他清晰的条目中代表。列出的机构是 对于中心，这不一定是调查员的机构。 肌酶临时访问计划（MTAP）是为患者提供庞贝疾病 美国获得静脉酶替代疗法（ERT）的机会 在有限的时间内扩展制造过程，直到批准以此规模的生产为止 FDA的商业用途。 通过此扩展过程产生的肌酶已在 迄今为止的大量临床研究，包括正在进行的临床试验 庞贝病（AGLU02704）（CNMC是该试验的一个部位），所有商业患者 在美国以外。 与重组蛋白质制造通常一样，该过程是 以较小的体积开发，优化并随后扩展以产生较大的 卷。 Genzyme正在寻求根据规模制造制造的产品的许可 通过对肌酶现有的生物制剂申请申请的补充进行处理。 这 协议，允许需要大剂量肌酶的成年患者接受这一大规模 制造产品还将确保足够的FDA批准的较小量 产生的肌酶用于婴儿和幼年患有pomoe疾病的患者。