FDA Rescinds Two Breakthrough Therapy Designations

Regulatory NewsRegulatory News | 08 March 2019 |  By 

In a rare move, the US Food and Drug Administration (FDA) this month has rescinded two companies’ breakthrough therapy designations (BTDs) because of clinical trial data.

For Tonix Pharmaceuticals, FDA informed the company recently that the BTD granted in December 2016 for Tonmya (cyclobenzaprine HCl), which is in Phase 3 development for the treatment of post-traumatic stress disorder (PTSD), has been rescinded because interim analysis data on Tonmya from the HONOR study did not support the continuation of the BTD. The company halted the study last July “due to inadequate separation from placebo on the primary endpoint at week 12.”

Seth Lederman, president and CEO of Tonix, said in a statement: “The rescission of Breakthrough Therapy designation of Tonmya for PTSD does not alter our NDA plan and will have minimum impact on our future interactions with the FDA. We are on track to start imminently the Phase 3 RECOVERY trial in civilian and military-related PTSD.”

Similarly, this week, based on FDA’s review of data from Trevena’s Phase 3 studies of oliceridine, which is intended for the management of moderate to severe acute pain, FDA informed Trevena that under the conditions studied, “these data were not sufficient to support the continuation of FDA’s previously granted Breakthrough Therapy designation.”

Last November, FDA issued a complete response letter for oliceridine, with FDA requesting additional clinical data and indicating that the submitted safety database is not of adequate size for the proposed dosing.

Trevena said it does not expect the absence of the BTD to impact the timing of FDA’s review of its resubmitted oliceridine new drug application.

The two rescinded BTDs in the same month is rare. FDA’s Center for Drug Evaluation and Research in all of 2018 only rescinded four BTDs, while in 2017, only three were rescinded. And FDA’s Center for Biologics Evaluation and Research has never rescinded a BTD.

In explaining why some applications lose their BTD status, FDA said in guidance on expedited programs for serious conditions from 2014: “Not all products designated as breakthrough therapies ultimately will be shown to have the substantial improvement over available therapies suggested by the preliminary clinical evidence at the time of designation. If the designation is no longer supported by subsequent data, FDA may rescind the designation. Because FDA commits significant resources to work particularly closely with sponsors of breakthrough therapy products, the Agency needs to focus its resources on breakthrough therapy drug development programs that continue to meet the program’s qualifying criteria.”

And FDA said it will offer sponsors “an opportunity to justify its product’s continued designation. FDA recognizes that sponsors of products that have had their breakthrough therapy designation rescinded because available data no longer support the designation may still have sufficient evidence after completion of the drug development program to support marketing approval.”


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