Had a read through the article. You should watch the JP webcast...

Had a read through the article.

You should watch the JP webcast as I think he answers your questions. He made it clear that because of the severity of the condition, it is challenging to actually come up with outcome measures that will indicate if the drug works or not.

Amendments to the phase-3 trial were also organised with the FDA. The FDA specifically wanted a longer trial in order to scrutinize the medicine further against placebo. If you compare the placebo and the drug efficacy over time, they actually start to have an even wider statistical difference as time goes on, indicating the drug is working. This is what the FDA wanted to see.

"However, nothing is ever a slam dunk with the FDA for NDAs for a multitude of reasons. And this is regardless of Orphan Drug designation, priority reviews, high unmet patient needs, or if there’s no other approved treatments for the indication etc etc etc."

It actually does matter. Accelerated Approval Program - The FDA instituted its Accelerated Approval Program to allow for earlier approval of drugs that treat serious conditions, and that fill an unmet medical need based on a surrogate endpoint.

The FDA approves NDAs, even if the clinical benefit is unknown if it fills an unmet need and the condition is serious and it shows changes in biomarkers (or another measure) that are associated with the condition...NEU's PH3 endpoints aren't surrogate. They are subjective outcome measures that already demonstrate clinical benefit. The FDA has approved drugs that have shown no clinical benefit in hope that through the accelerated program the company can gather more data on what benefits the drug is providing to patients.

Obviously nothing is ever 100%, but if there was ever going to be a slam dunk with the FDA, this is it.