Breakthrough therapy designation?I'm not sure how important getting such a designation is, but I see that Novo Nordisk buried it in a footnote that they received breakthrough therapy for semaglutide in NASH. Recall this is their existing diabetes drug and they want to see how it works in liver fat and fibrosis. They just finished a Phase 2 test that showed it hit the NASH endpoints for most patients. It looks to be a decent drug.
As Qwerty pointed out, it's along the same lines as tesamorelin in that it involves the IGF-1 pathway (don't ask me, ask him on the science). As we've heard before, a likely interested big player in what THTX is doing would be Novo Nordisk.
F8 has come along in a somewhat similar way -- an existing drug for an indication seeing how it performs in NASH. Now Novo had clear Phase 2 data that showed it worked and we have the HIV NAFLD data and the "totality" of all the various studies done on tesamorelin. I wonder if THTX may also get breakthrough therapy designation. From what they have on the website, it would have seemed semaglutide wouldn't have been a breakthrough as it wasn't (as far as NASH usage) in the original NDA for diabetes. So I'm not sure what that means, but it would mean you can't rule it out for GRGH being breakthrough as far as NASH either. Frankly, not sure what economic benefit it affords and accellerated approval is the one thing that confers a bit of value and "credibility". As Harrison said in the conference call, accellerated is still all available for any NASH drug and I would hope they get it given the safety.
From FDA website: A drug that receives Breakthrough Therapy designation is eligible for the following:
- All Fast Track designation features
- Intensive guidance on an efficient drug development program, beginning as early as Phase 1
- Organizational commitment involving senior managers
Ideally, a Breakthrough Therapy designation request should be received by FDA no later than the end-of-phase-2 meetings if any of the features of the designation are to be obtained. Because the primary intent of Breakthrough Therapy designation is to develop evidence needed to support approval as efficiently as possible, FDA does not anticipate that Breakthrough Therapy designation requests will be made after the submission of an original BLA or NDA or a supplement. FDA will respond to Breakthrough Therapy designation requests within sixty days of receipt of the request.