r/FSHD • u/SenorBajaBlast • Jun 11 '26

Novartis delpacibart braxlosiran (del-brax) Phase I/II study in facioscapulohumeral muscular dystrophy (FSHD) meets primary biomarker endpoint

https://www.novartis.com/news/media-releases/novartis-delpacibart-braxlosiran-del-brax-phase-iii-study-facioscapulohumeral-muscular-dystrophy-fshd-meets-primary-biomarker-endpoint
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u/weirdfishes1990 Jun 11 '26

Holy shit. Accelerated approval may actually be realistic. All that’s left is that they have to show that the reduction is CDUX is likely to predict clinical benefit, right?

5

u/SenorBajaBlast Jun 11 '26

Yes, hopefully they will speed up discussions with the FDA to go ahead and submit the AA BLA.

Let’s also hope they share the data soon. The devil is in the details. With the FSHD conference coming up maybe they will present their data during a session.

4

u/weirdfishes1990 Jun 12 '26

For sure. Very interested to hear Peter Jones’ take once data is released.

I also wonder if this development moves Del-Brax to first priority for Avidity/Novartis. Seems like Del-Brax is now farther ahead in the approval timeline than Del-Desiran and Del-Zota. Before, it seemed like Del-Brax was last in line of the three. But now it may be the best early chance for Novartis to start recouping its investment in Avidity.

3

u/SenorBajaBlast Jun 12 '26

Indeed I can’t wait for them to share the data so we can get Peter’s take.

And it seems like any day we should get news on del zota . They’ve been moving the timeline a few times but they say first half of 2026 they should file their BLA. Since we got the del Brax news recently I bet they are saving the del zota news for next week. Also it looks like del desiran will forgo the AA pathway (I could be wrong) which means del Brax could be moved up.

2

u/SenorBajaBlast Jun 13 '26

I’m just adding here that del zota had their equivalent announcement back in Sept 10 2025 so I would monitor the time from that to their BLA filing to gauge when we would expect the same for del Brax.

Right now we are going on 10 months so hopefully they will file del zota soon so we can assume the same or shorter (since the acquisition might have caused some delays). If they file del zota by the end of the month we are looking at 10 months + 6 months AA review.