Breakthrough therapy designation conveys all of the fast track program features… more intensive FDA guidance on an efficient drug development program, an organizational commitment involving senior managers, and eligibility for rolling review and priority review…. Given that the primary intent of breakthrough therapy designation is to provide timely advice and interactive communications to help the sponsor design and conduct a drug development program as efficiently as possible, including the potential use of alternative trial designs, the full benefits of breakthrough therapy designation can only be realized during the development program, well in advance of the submission of the original BLA or NDA, or a supplement.
A sponsor of a drug that receives fast track designation will typically have more frequent interactions with FDA during drug development. In addition, products that have been designated as fast track can obtain rolling review.
Any drug, including those that have received a fast track designation (or) breakthrough therapy designation… can be granted priority review, if the relevant criteria are met.
As it stands, as a designated orphan drug indicated for a severe disease of unmet need, with fast track designation, NNZ-2591 in PMS will be eligible for more frequent interactions with the FDA during development, a rolling review (if relevant) and priority review ( 6 month NDA review rather than ~10 months). If the Rare Pediatric Disease Priority Review Voucher Scheme is reinstated, it will also be eligible for consideration for a tradeable Priority Review Voucher.
Thus, post-Phase 3, BTD is likely to confer limited benefit in terms of acceleration to approval. It’s probably fair to say that the biggest impact would be on perception – it has been shown that linking the word “breakthrough” to a drug leads to the common perception that the drug has superior effectiveness and stronger supporting evidence. This, of course, may not be reflect reality.
Perceived validation of superiority of the drug by investors can also positively impact share price, especially if the drug has been developed by a smaller biotech rather than a large pharma.
One example of this is Acadia’s Nuplazid. When BTD was granted to Nuplazid in 2014, the ACAD shareprice showed a one day increase of 13.80% and a 7-day increase of 10.5%.
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