USA – Regenerative Medicines: FDA Finalizes Guidance on Expedited Programs

With almost 80 regenerative medicine advanced therapy (RMAT) designation requests in 2017 and 2018, it’s clear that the US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) has been busy with RMATs, though only on Friday did the agency finalize guidance from 2017 on the designation.

Advantages of the RMAT designation, according to the guidance, include all the benefits of the fast track and breakthrough therapy designation programs (sponsors may apply for and receive more than one designation for a given product), including early interactions with FDA. Although, as opposed to breakthrough therapy, the RMAT designation “does not require evidence to indicate that the drug may offer a substantial improvement over available therapies,” the guidance explains.

FDA spells out precisely what constitutes a therapy that could qualify for an RMAT designation, which includes cell therapies, therapeutic tissue engineering products, human cell and tissue products and combination products using such therapies…