Wow! This could be a pretty big deal for Neuren and NNZ-2591....

Wow!
This could be a pretty big deal for Neuren and NNZ-2591. Its a surprising shift- Prasad is now saying the FDA will act "at the first sign of promise" for rare disease drugs — even if they haven’t fully proven traditional efficacy yet.He’s talking about fast-tracking approvals using surrogate endpoints (things like biomarkers or early clinical signs) and relying more on real-world data to monitor how drugs perform once they’re actually being used. This kind of flexibility could really help a company like Neuren, which, as we know, works in ultra-rare neurodevelopmental disorders where running large randomized controlled trials is very difficult.Prasad’s stance lines up with comments made earlier by FDA Commissioner Marty Makary, who outlined a new pathway where scientifically plausible therapies for rare diseases might get conditional approval based on small or single-arm trials. The idea is to give patients early access to potentially helpful treatments, and then keep monitoring safety and effectiveness in the real world.This is a bit of a turnaround for Prasad. Before joining the FDA, he was vocal about not liking the low approval bar for some rare disease drugs, especially gene therapies. So investors were initially spooked when he was appointed. But his latest comments suggest a more pragmatic, patient-focused approach — one that could actually be great news for biotech companies developing treatments for underserved conditions.For Neuren, the takeaway is that there’s now potentially a much smoother path to get NNZ-2591 to market — especially if the drug shows early positive signals and there's strong unmet need. Even if traditional trials are hard to run or take a long time, the FDA might still be open to getting the drug out there earlier, then tracking how it performs in practice.Neuren could position itself really well for early approvals or partnerships. This might also shift market sentiment, especially if investors start to see the FDA as a genuine ally in rare disease drug development rather than a hurdle.All up, this could help accelerate the timeline for NNZ-2591 and give Neuren a real edge .