Where to in 2021?, page-9

The timing seems reasonable to me.

I’d expect that Acadia will request a pre-NDA meeting with the FDA. The pre-NDA meeting should occur no less than 2 months prior to NDA submission to allow for a meaningful response to FDA feedback. There is also up to a 2 month wait for the meeting from time of request.

Then there’s the job of compiling the NDA. Compiling an NDA is complex and time-consuming. It is a comprehensive document with 15 sections, typically 100,000 pages long. You need to get it right or else the FDA will refuse to accept it for filing.

If you look at how it went with Acadia’s last submission of a marketing application (a supplementary NDA for pimavanserin), following the announcement of Phase 3 results in early December 2019, the sNDA was filed in late July 2020 (so, 7-8 months).

Not only does a mid-2022 filing seem reasonable to me, I am also very confident that Acadia will hit the ground running on this. With almost 6 years elapsed since their first and only marketing approval, they badly need this marketing approval as well.

For those worried about trial participants and their access to trofinetide, 95% of those who completed the main trial (LAVENDER) elected to enrol in the 9 month extension study (LILAC). After that, those eligible are able to continue treatment for more than 2.5 years in the LILAC-2 extension trial. That should see them through until anticipated approval. I’d expect that this will mean continued access for most patients, as the Acadia CSO said in the conference call this week that there had been zero drop-outs from the LILAC trial on account of adverse events (which is also an excellent sign that diarrhoea management programs were able to be successfully implemented during the less-protocol bound extension trial).

https://rettsyndromenews.com/2021/1...inetide-lavender-phase-3-trial-top-line-data/