Hey Steini, great post and I think a really important point...

Hey Steini, great post and I think a really important point you’ve made around treatments not being ideal, but still beneficial. A bit of an apples to oranges case, however I think aptly explains the paradigm shift of FDA thinking for new and cutting edge therapies is the recent Sarepta Accelerated Approval and now Traditional Approval of their drug treating Duchenne Muscular Dystrophy (DMD). While the drug didn’t meet it’s primary endpoint and has a slew of issues in their CMC package, it was still given approval by the FDA. I don’t necessarily agree with their approval because I do think there are a lot of things left wanting in their total package, but DMD is a rare and devastating disease in which there is no cure so a treatment that shows the prospect of direct benefit with a decent safety profile can be accepted (plus they had a very outspoken patient advocate on the advisory panel). Again, apples to oranges given this disease has great unmet need, however I think an interesting case in which the bar is modified to give patients with no previous option one to consider.

I am NOT advocating that sub-standard therapies receive approval, just shining light on interesting scenarios for others to inform their own opinions.

https://www.advisory.com/daily-briefing/2024/06/25/sarepta-approval