UniQure NV (QURE, Financial) saw its stock surge over 121% following the FDA's favorable decision regarding its regenerative medicine advanced therapy (RMAT). The FDA agreed to use data from the ongoing Phase I/II study, compared with external natural history controls, as a primary basis for a Biologics License Application (BLA) under the accelerated approval pathway. This eliminates the need for additional pre-submission studies.
The FDA also approved the use of cUHDRS as an intermediate clinical endpoint. Furthermore, the reduction of neurofilament light chain (NfL) levels in cerebrospinal fluid (CSF) can serve as supportive evidence of treatment efficacy in the accelerated approval application.
