UniQure Reaches Agreement With FDA On Key Elements Of An Accelerated Approval Pathway For AMT-130
UniQure Reaches Agreement With FDA On Key Elements Of An Accelerated Approval Pathway For AMT-130
As part of uniQure's Regenerative Medicine Advanced Therapy (RMAT) Type B meeting held in late November, the FDA agreed that data from the ongoing Phase I/II studies, compared to a natural history external control, may serve as the primary basis for a BLA submission under the Accelerated Approval pathway, avoiding the need for an additional pre-submission study.
作为uniQure的再生医学先进疗法（RMAT）Type b会议的一部分，FDA同意将正在进行的I/II期研究的数据与自然历史外部对照进行比较，可以作为加速批准路径下提交BLA的主要依据，从而避免额外的预提交研究。
The FDA also agreed that cUHDRS may be used as an intermediate clinical endpoint and that reductions in neurofilament light chain (NfL) measured in cerebrospinal fluid (CSF) may serve as supportive evidence of therapeutic benefit in the application for accelerated approval.
FDA还同意将cUHDRS作为中间临床终点，并且在脑脊液（CSF）中测量的神经纤维轻链（NfL）的减少可以作为加速批准申请中治疗效果的支持证据。
