Y-mAbs plans to submit clinical/safety, non-clinical portions of BLA in November
Y-mAbs Therapeutics announced that it has completed a Type B Pre-Biologics License Application meeting with the U.S. Food and Drug Administration regarding a potential pathway for FDA approval of naxitamab for the treatment of patients with relapsed/refractory high-risk neuroblastoma. At the meeting, the Company reached alignment with the FDA on an Accelerated Approval Pathway for naxitamab along with a rolling BLA submission. The Company expects to submit the Clinical/Safety portion and the non-Clinical portion of the BLA in November 2019. For the CMC portion, the Company believes it will have sufficient data from the process performance qualification batches to complete the CMC portion in early 2020. However, Y-mAbs is still investigating possibilities for accelerating the submission of the CMC portion, and hope to comply with the FDA requirements at an earlier time. Under naxitamab's breakthrough therapy designation, the compound qualifies for a Rolling BLA, which enables individual modules of the application to be submitted by the Company and reviewed by the FDA on a rolling basis, rather than waiting for all sections to be completed before submission. The rolling application process will provide the Company with the opportunity for ongoing communications with the FDA, and, during this rolling process, the Company anticipates that it will be able to address any substantial matters raised by the FDA. Based on the previously announced efficacy data from Study 12-230 in relapsed/refractory high-risk neuroblastoma patients at Memorial Sloan Kettering Cancer Center, the FDA determined that efficacy data from all 37 patients of the Company's multicenter Study 201 would not be required for the BLA filing. The FDA advised the Company that the available data for the first group of patients treated outside MSK in Study 201 would be sufficient for the BLA filing. The first group consists of 24 patients, of which 11 were evaluable prior to the pre-BLA meeting and showed an overall response rate of 73%, including 55% complete responses, as assessed by the investigators. The Company intends to announce the complete dataset for Study 201 once the data becomes available.