Unfortunately “better than even chance” wasn't good enough, as the FDA ruled that Neuren's preliminary evidence wasn't sufficient “at this time”. It's a disappointing outcome for Rett families, Neuren and its shareholders.
The purported aim of FDA breakthrough therapy designation is to assist those drugs which show promise of significantly improving outcomes in serious conditions to reach patients with unmet needs as soon as possible. 'Streamline drug development and regulatory review” in the words of Neuren's announcement.
The FDA issued a guidance document last May on the use of its four expedited drug approval programs, including breakthrough product designation. In the final guidance, the FDA defined the "preliminary clinical evidence" required for BTD. This was said to mean "evidence that is sufficient to indicate that the drug may demonstrate substantial improvement in effectiveness or safety over available therapies, but in most cases is not sufficient to establish safety and effectiveness for purposes of approval." Such evidence needed to indicate "a substantial improvement over available therapies." Drugs intended to treat a disease for which there was no currently available standard of care were confirmed to also be eligible for the designation. According to the FDA, the necessary preliminary evidence could generally be obtained from Phase I or II trials
Well that's the FDA stated aim and guidance. Then there's the reality of the program in action.
On Saturday BTD designation was announced for TaiMed Biologics’ ibalizumab for the treatment of HIV/AIDS infection. I admit I was dismayed when I read of ibalizumab's long development path leading to BTD.
It is now more than 12 years since Phase 1 results for the drug were announced. It is 11 years since the drug was granted fast track status by the FDA. Phase 2a trials were completed in 2006. A 113 patient, one-year long Phase 2b trial was completed in 2011. A further bridging trial was completed in 2012. Late stage trials are due to be completed in 2017. At best, even with breakthrough therapy designation, the clinical phase of development for ibalizumab prior to approval will have taken at least 15 years.
It's difficult to know what to say, other than that there is clearly no danger that the term “breakthrough” with respect to FDA breakthrough therapy designation could ever be confused with “breakneck', as in speed.
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