Question about expanding the Afrezza label for pediatric use

[mnholdem]

Perhaps there are some experts on ProBoards who can answer this question (that would be preferred to speculation). MannKind's CMO David Kendall MD recently disclosed that the company has complete the 1st cohort (ages 14-17) of their active pediatric clinical trial to expand Afrezza's label. 

Here's the specific question:

• Is MannKind (or any company) allowed to submit an sNDA to the FDA as soon as primary outcomes have been assessed/validated for a specific cohort and then submit addition applications for additional cohorts when the trial data is complete for each?

I'd sincerely appreciate any ProBoard members have experience in this matter, or know of a trial where this was allowed, to weigh in.

Thank you!

[matt]

In general, it is up to the sponsor to decide what to submit and when. The agencies have gotten a little bit tougher on mandatory pediatric trials when a medication tested on adults might be used off-label in an unintended and untested population. In principal, there is no reason to disallow expanding the label gradually but FDA may not allow it because:

A. They want a proper pediatric trial and by gradually sneaking up on younger age groups it may increase the rate of off-label usage.
B. There is still the matter of long-term safety data. Safety in young persons just reaching puberty, but who have immature bodies, may be an issue.

I would expect that FDA will want to see a lot more than primary outcomes, but if they are in a generous mood it might slide through. There is a lot of wiggle room on either side.

[buyitonsale]

JDRF is under pressure also to keep donations coming... let's see them help MNKD and themselves by putting some pressure on FDA.

Yes, parents and children want inhaled insulin!