Breakthrough Therapy Designation is Not Permanent: FDA Guidance Emphasizes
(Thursday, June 30, 2022)
In one of its shortest guidance documents, FDA clarified that the highly coveted and tough to get Breakthrough Therapy Designation (BTD) can be rescinded for multiple reasons. The description of the conditions for rescinding BTDs are not new and have been defined in previous FDA documents, so the purpose of this new guidance is not clear except for FDA to indicate its desire to take BTDs away more aggressively from developers whose drugs show fewer promising results post the initial ones that led to the BTD in the first place. Among all the expedited approval pathways granted by the FDA, the BTDs have been the most coveted by developers as they validate the promise of the preliminary results in early clinical trials, and many of the BTD products got FDA approval in a much shorter time. The financial reward of the BTD is debatable but most small companies receiving BTDs were attractive targets for acquisition by big pharma which may translate into better returns for the initial developers. In any case, the rescinding of the BTD is bad news for the owners of these designations. The guidance lists three conditions for FDA to rescind a BTD: (1) another drug got approved for the same indication, (2) Subsequent clinical trials did not yield equally promising results, or (3) the developer did not pursue development of the BTD drug as indicated by the lack of progress reports. These conditions have been described in the previous guidance documents and FDA’s MAPPs, so should not come as a surprise to anyone. Perhaps that’s the reason the guidance is less than two pages long. However, it does emphasize once again to anyone with a BTD drug, getting the BTD is just the first step in the continual aggressive perusal of the drug, to be verified by the FDA, failing which, the incentives for the BTD could be taken back by the FDA. Not news, just facts.
Dr. Mukesh Kumar
Founder & CEO, FDAMap
Linkedin: Mukesh Kumar, PhD, RAC