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Thanks RYN for drawing my attention to a potential DMD inflammation treatment in very late stage drug development. I’d ALSO like thoughts from those with more medical knowledge, but will outline some thoughts and questions from an initial dive, no guarantee of value-add though.

1. Italfarmaco’s drug, givinostat, has met endpoints in phase 3 trial for DMD. The FDA has accepted their NDA and set a PDUFA date (deadline to complete their review and either approve, deny, or ask for more to be done) of Dec 21, 2023. Italfarmaco used a PRV to get the review date 4 months closer (10 down to 6 mos).

2. Phase 3 trial was on 179 late stage ambulatory boys (mean age 9). I suspect this was to make their primary endpoint testing consistent (time taken to walk up 4 stairs). Apart from no longer being able to walk, I don’t think there is any medical change in inflammation or muscular dystrophy mechanism between ambulatory and non-ambulatory DMD, albeit it (inflammation) is gradually more prevalent as age increases.

Creates two other questions:

(1) If approved, can the treatment be prescribed and reimbursed to non-ambulants as well?

(2) Is there any difference between ambulant and non-ambulant boys as far as private insurer reimbursement goes? I understand US govt reimbursement (Medicaid) is mandatory once FDA approves.

3. ANP left it out of their corporate preso outlining competition. Why? They included other ambulant treatments in development.

4. Givinostat did not get accelerated approval after phase 2. That might be because they targeted ambulant boys.