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RMAT Designation Requests May Rise Or Fall On Manufacturing Changes, Clinical Data Breadth

 
• By Sue Sutter

Product characterization questions and partial or inconsistent clinical data are among the more common reasons why the US FDA denies, or seeks additional information about, a request for regenerative medicine advanced therapy designation, FDA’s Bryan says; sponsors should consider why they are seeking designation when deciding whether to apply for RMAT, breakthrough therapy or both.

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Whether FDA says 'yes' or 'no' to an RMAT designation request may depend upon changes to the product during development and the extent of clinical data provided on different outcomes.

Product characterization questions and partial or inconsistent clinical data are among the more common reasons why the US FDA denies, or seeks additional information about, a request for regenerative medicine advanced therapy (RMAT) designation, according to the head of the office that oversees the expedited pathway.

Manufacturing remains a focus of concern during the intensive interactions between the agency and sponsor upon receipt of RMAT designation, while attention on clinical issues switches to appropriate endpoints and trial design, Office of Tissues and Advanced

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