Back in March 2015 their application for BT designation for Trofinetide to treat Retts was denied. From the annoucement at the time- "However, Breakthrough Therapy will not be granted at this time because the FDA considered that data from Neuren’s exploratory Phase 2 trial provide insufficient evidence to demonstrate substantial improvement over existing therapies using conventional statistical methods"
The P2 trial that preceded the BT application was in 53 sujects aged 16 to 45 years of age. From the November 2014 annoucement- "The probability of observing this combined degree of clinical benefit both in the group-level and subject-level analyses and no clinically significant worsening in any endpoint purely by chance (the “false-positive” rate) was determined as 2.3% (p=0.023) by permutation testing. In the permutation testing, randomly simulated allocations of patients to NNZ-2566 and placebo were repeated 1000 times and positive outcomes in the data were counted. The statistical approach used in the analysis of the trial results, including permutation testing, has been published in a peer-reviewed journal and used in previous trials in other therapeutic areas, including for the development of a product recently granted Breakthrough Therapy designation by the FDA"
Im not sure if the additional data the FDA has requested in relation to PM is to support their BT application or not but i am certain they would have applied for it.
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