FDA gives advanced tag for Magenta cell therapy for inherited metabolic disorders

The FDA designates Magenta Therapeutics’ (MGTA +1.3%) one-time cell therapy MGTA-456 a Regenerative Medicine Advance Therapy (RMAT) for the treatment of multiple inherited metabolic disorders.

RMAT is akin to Breakthrough Therapy status for drugs/biologics. It provides for the accelerated review of the marketing application and more intensive guidance on development.

Phase 2-stage MGTA-456 is designed to stop the progress of the metabolic disorders by providing a high dose of stem cells that regenerate a new immune system in the patient.

https://seekingalpha.com/news/3496769-fda-grants-rmat-tag-magenta-cell-therapy-inherited-metabolic-disorders