Definition
RMAT (Regenerative Medicine Advanced Therapy) is an FDA designation created by the 21st Century Cures Act (2016) to expedite development and review of regenerative medicine products that treat, modify, reverse, or cure serious conditions. It applies to cell therapies, gene therapies, therapeutic tissue products, and related combination products.
How RMAT Works
Sponsors request RMAT designation with preliminary clinical evidence showing the regenerative medicine may address unmet needs in serious conditions. FDA provides enhanced engagement on development and manufacturing.
Eligibility Criteria
- Regenerative medicine product (cell, gene, tissue, or combination)
- Intended to treat, modify, reverse, or cure serious condition
- Preliminary clinical evidence of potential benefit
- Addresses unmet medical need
RMAT Benefits
RMAT provides all Breakthrough Therapy benefits plus:
| Benefit | Description |
|---|---|
| Early Manufacturing Discussions | FDA engagement on CMC before Phase 3 |
| Rolling Review | Submit BLA sections as completed |
| Priority Review Eligibility | 6-month review goal |
| Accelerated Approval Eligibility | Surrogate endpoint approval |
| Post-Approval Flexibility | Potential for conditional approval |
Why BD Teams Track RMAT
For business development professionals, RMAT signals advanced therapy innovation:
- Deal Implication: RMAT assets in gene/cell therapy space command premium valuations
- Due Diligence Focus: Assess manufacturing complexity and scalability for commercial production
- Opportunity Signal: RMAT companies often need manufacturing and commercialization partners