Breakthrough Therapies: Failed Study Won’t Automatically Lead To Rescinded Designation
The decision to maintain or revoke breakthrough therapy status when trial results do not appear to support the original designation will depend on the facts specific to a development program, the US FDA says in new draft guidance. Since 2012, the agency has granted 475 breakthrough therapy designation requests and rescinded 35 designations.
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Breakthrough Therapies: Another Drug’s Approval Should Not Lead To De Facto Rescission, Industry Says
Industry and patient advocacy groups urge the agency to consider a variety of factors in determining whether a drug’s breakthrough designation should be rescinded upon the approval of another product; draft guidance language on quality of clinical evidence to be considered in rescission decisions raises concerns specific to rare disease populations.
Popular expedited review program results in a higher approval rate than the related fast track designation, but the COVID-19 era is seeing a drop in designations and a rise in complete response letters for BTD programs.
US FDA rescinds breakthrough designations for Tonix’ PTSD therapy Tonyma and Trevena’s novel pain drug Olinvo after clinical trial disappointments – and after identifying other regulatory pathways to market.