Under Section 3033 of the 21st Century Cures Act, a drug is eligible for regenerative medicine advanced therapy (“RMAT”) designation. To obtain this designation, the treatment must be “a cell therapy, therapeutic tissue engineering product, or any combination product using such therapies or products, “intended to “treat, modify, reverse, or cure a serious or life-threatening disease” and have preliminary clinical evidence that the drug has the potential to address unmet needs. In its Guidance for Industry entitled “Expedited Programs for Serious Conditions-Drugs and Biologics,” the FDA has defined its interpretation of whether a disease or condition is serious or life-threatening and whether a drug is intended to treat a serious disease or condition.

Copyright: kentoh / 123RF Stock Photo
Copyright: kentoh / 123RF Stock Photo

The RMAT designation provides an easier path to approval by lowering the bar for unmet medical need for serious or life-threatening diseases. The request for RMAT designation must be made either concurrently with submission of an Investigational New Drug (“IND”) application or as an amendment to an existing IND.  Additional data other than that required for an IND will not be required for a RMAT request.

This designation will provide additional resources from the FDA to expedite the approval process.  The agency will take an active role in assisting the applicant with advice during review.

Time will tell how well this provision is executed under the new law. For now, we have our first publicly disclosed RMAT designation for Humacyte and its product Humacyl, announced in March.