The designation follows durable responses seen in a phase 1 study of the agent.
The hematologist/oncologist from Mayo Clinic discussed the role of HLA loss in a variety of cancers.
The investigational gene therapy, which will receive priority review, has a PDUFA date of May 20, 2022.
From baseline, all participants showed improvements on CGI-Improvement scale scores while all but 1 patient demonstrated improvement on modified Hoehn and Yahr scores.
The recommended phase 2 dose of cilta-cel elicited deep and durable responses, along with a tolerable safety profile.