Regenerative Medicine Therapies: Manufacturing Changes May Not Impact RMAT Designation
Final guidance notes that preliminary clinical evidence may come from studies conducted outside the US, says historical controls may be considered in clinical trial designs.
You may also be interested in...
Rejection of an RMAT request does not mean necessarily mean the FDA won't grant one. The agency will accept new clinical data to support the application through a “reconsideration” process, industry official says.
Fresh Or Frozen? Orchard Aims For Cryopreserved Gene Therapy Approvals Using Pivotal Trials With Fresh Cell Formulations
Orchard aims to submit three ex vivo autologous gene therapies for approval by the end of 2021, helped by a new regenerative medicine advanced therapy designation (for a rare primary immunodeficiency syndrome) and a breakthrough therapy designation (for another rare primary immunodeficiency).
Requiring that certain chemistry, manufacturing and controls conditions be satisfied before awarding breakthrough or regenerative medicine advanced therapy status would help ensure that the quality side of product development keeps pace with the clinical side, says Parexel's Mo Heidaran, a former CMC reviewer in the FDA’s biologics center.