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.
作爲uniQure的再生醫學先進療法（RMAT）Type b會議的一部分，FDA同意將正在進行的I/II期研究的數據與自然歷史外部對照進行比較，可以作爲加速批准路徑下提交BLA的主要依據，從而避免額外的預提交研究。
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還同意將cUHDRS作爲中間臨床終點，並且在腦脊液（CSF）中測量的神經纖維輕鏈（NfL）的減少可以作爲加速批准申請中治療效果的支持證據。
