Afrezza Safety and Pharmacokinetics Study in Pediatric Patie

[harryx1]

Afrezza Safety and Pharmacokinetics Study in Pediatric Patients

www.clinicaltrials.gov/ct2/show/NCT02527265?term=diabetes&lup_s=08/04/2015&lup_d=14&show_rss=Y&sel_rss=mod14

[centralcoastinvestor]

Thanks for posting this.  I have a couple of observations.  First, I noticed that the study says it is a Phase 1 study.  I would have thought this would have been a Phase 3 study since Afrezza has already been has been extensively studied.  Also, with an end date out in July 2017, it sure seems like Sanofi is very much committed to Afrezza.

[liane]

Children are not small adults. Afrezza has not been trialed in the pediatric population. Phase I required for safety and to establish dose range in this population.

I would add, that SNY doing this study shows huge commitment. So many drugs are never trialed in kids. This is huge!

[brentie]

If the phase 1 trial takes 2 years, the kids that want it today will be retired by the time it's approved. 

[johnhindepost]

Nice one Harry,  you deserve another star!

[brentie]

Aug 18, 2015 8:45:27 GMT -5 liane said:

Children are not small adults. Afrezza has not been trialed in the pediatric population. Phase I required for safety and to establish dose range in this population.

I would add, that SNY doing this study shows huge commitment. So many drugs are never trialed in kids. This is huge!

Liane,I don't want to rain on your parade but wasn't  this trial required  by the FDA for approval?


The FDA is requiring 4 postmarketing studies for Afrezza:



A clinical trial to evaluate pharmacokinetics, safety and efficacy in pediatric patients.



A clinical trial evaluating the potential risk for pulmonary malignancy with Afrezza, along with assessment of cardiovascular risk and long-term effect on pulmonary function.



2 pharmacokinetic-pharmacodynamic euglycemic glucose-clamp clinical trials, 1 characterizing dose-response, the other within-subject variability.


www.medscape.com/viewarticle/827539

[mnholdem]

Aug 18, 2015 9:22:25 GMT -5 brentie said:

Aug 18, 2015 8:45:27 GMT -5 liane said:

Children are not small adults. Afrezza has not been trialed in the pediatric population. Phase I required for safety and to establish dose range in this population.

I would add, that SNY doing this study shows huge commitment. So many drugs are never trialed in kids. This is huge!

Liane,I don't want to rain on your parade but wasn't  this trial required by the FDA for approval?

The fact that it's required is not relevant, IMHO. Sanofi could have easily delayed. The fact that they haven't put this off PLUS they have moved up the timeline for the trial completion date tells me that Sanofi has no intention of dropping Afrezza, contrary to short interest claims being published in hit pieces.

Also, the link that harry provided is, I believe, the updated one with the protocols that have been negotiated between the FDA and Sanofi/MannKind. As such, the trial may contain a few additional endpoints to clarify superiority and improve labeling down the road. By then, I suspect that the label will have gone through a few revisions, though.

Something tells me that Sanofi wants these post-marketing trials and other studies done and published ASAP. 

[sportsrancho]

For what ever it's worth. Afrezza seems to be acting exactly the same way with kids. My client is in touch with other parents with kids on it. As the word spreads the parents will get their T1 kids on it ASAP! Off-label.

[notamnkdmillionaire]

Aug 18, 2015 12:37:45 GMT -5 sportsrancho said:

For what ever it's worth. Afrezza seems to be acting exactly the same way with kids. My client is in touch with other parents with kids on it. As the word spreads the parents will get their T1 kids on it ASAP! Off-label.

That's great news SR. T1s are paving the way but AFrezza really needs the T2s to make it successful.

[sportsrancho]

I think the hardest sell is getting the older ones that think they have it controlled with Metformin to try it. The Doc's will need to push this. But only a matter of time. The FlexPen is dead!!

[liane]

Aug 18, 2015 9:22:25 GMT -5 brentie said:

Aug 18, 2015 8:45:27 GMT -5 liane said:

Children are not small adults. Afrezza has not been trialed in the pediatric population. Phase I required for safety and to establish dose range in this population.

I would add, that SNY doing this study shows huge commitment. So many drugs are never trialed in kids. This is huge!

Liane,I don't want to rain on your parade but wasn't  this trial required  by the FDA for approval?


The FDA is requiring 4 postmarketing studies for Afrezza:



A clinical trial to evaluate pharmacokinetics, safety and efficacy in pediatric patients.



A clinical trial evaluating the potential risk for pulmonary malignancy with Afrezza, along with assessment of cardiovascular risk and long-term effect on pulmonary function.



2 pharmacokinetic-pharmacodynamic euglycemic glucose-clamp clinical trials, 1 characterizing dose-response, the other within-subject variability.


www.medscape.com/viewarticle/827539

brentie,

I don't think the wording of this Medscape report is quite accurate. While the other 3 studies are "required", it would have been better to say the pediatric studies are required if MNKD / SNY want the pediatric indication on the label.

[brentie]

Aug 18, 2015 16:11:33 GMT -5 liane said:

Aug 18, 2015 9:22:25 GMT -5 brentie said:

Liane,I don't want to rain on your parade but wasn't  this trial required  by the FDA for approval?


The FDA is requiring 4 postmarketing studies for Afrezza:



A clinical trial to evaluate pharmacokinetics, safety and efficacy in pediatric patients.



A clinical trial evaluating the potential risk for pulmonary malignancy with Afrezza, along with assessment of cardiovascular risk and long-term effect on pulmonary function.



2 pharmacokinetic-pharmacodynamic euglycemic glucose-clamp clinical trials, 1 characterizing dose-response, the other within-subject variability.


www.medscape.com/viewarticle/827539

brentie,

I don't think the wording of this Medscape report is quite accurate. While the other 3 studies are "required", it would have been better to say the pediatric studies are required if MNKD / SNY want the pediatric indication on the label.

I think it is accurate, Liane. Check out the FDA's press release...

There I go, raining again.


www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm403122.htm

[Deleted]

One would think a 2 unit cartridge will be necessary for the pediatric trial. If true, except an announcement next month.

[longstocking]

Aug 19, 2015 18:51:09 GMT -5 @kastanes said:

One would think a 2 unit cartridge will be necessary for the pediatric trial. If true, except an announcement next month.

That seems logical.  How hard could it be anyway?  They likely wouldn't need very many for a trial.

[liane]

Aug 19, 2015 18:51:09 GMT -5 @kastanes said:

One would think a 2 unit cartridge will be necessary for the pediatric trial. If true, except an announcement next month.

MNKD has a small batch line that they used for previous studies. They could dedicate this to producing 2-unit cartridges for the study without tying up any of the regular production lines.