BTD-2……..Set…… Musing 2

Photo by Jaime Cavazos

Photo by Jaime Cavazos

I am a researching junkie. True. Difficult to believe, I know. 😉 I like being “set”. I like knowing what I’m getting in for, whether it’s figuring out my daughter had Rett syndrome before any doctor would say “Your daughter has Rett syndrome” or my own neuromuscular disease before having that confirmed. I want to KNOW, be prepared, know the odds, be set.

When I signed Katie up for the NNZ trial, I reviewed the criteria, checked off all the inclusion criteria I knew she met, asked about others. By the time we went to Texas for baseline testing there were only two on the list I thought could go either way. I got blindsided when they said she might be excluded because she could talk. It was the only time in my life I hoped she couldn’t speak enough.

I also researched the heck out of NNZ -2566 and I stumbled upon the Army’s trial-Intrepid 2566. To me a long, on going trial meant safe. Check that off my list.

I found info about what it was, the theory behind how it works and even with my limited knowledge in science….I got it.


Did she likely meet the criteria? Check.

From what I could find (and it was a lot) was it safe? Check.

Did I believe the science behind it? Check.

Was I set to allow Katie to be in the trial? Oh, yes, I was.

And now there’s this Breakthrough Therapy Designation hanging over all of us. Is it the end of the line if we don’t get it? Absolutely, not. The overwhelming majority of drugs that get approved by the FDA don’t get this designation. The numbers tell you only 30% of applications for Breakthrough Therapy Designation are approved, and the number of applications is small.

But, I personally think the chances are better than that. Neuren has to prove two things, just two, to get it.

Taken from the FDA website:

On July 9, 2012 the Food and Drug Administration Safety and Innovation Act (FDASIA) was signed. FDASIA Section 902 provides for a new designation – Breakthrough Therapy Designation. A breakthrough therapy is a drug:
intended alone or in combination with one or more other drugs to treat a serious or life threatening disease or condition
preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.

If a drug is designated as breakthrough therapy, FDA will expedite the development and review of such drug. All requests for breakthrough therapy designation will be reviewed within 60 days of receipt, and FDA will either grant or deny the request.

Is Rett syndrome serious? Life threatening?? Unfortunately, yes. Check

Does Trofinetide (NNZ-2566) demonstrate substantial improvement over existing therapies? Well….since there aren’t any….I’m going to give that one a yes and check, too.

But, on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development?? I’m pretty sure the three Neuren showed in their results qualifies for “one or more”. Check

Do I believe that I saw things in Texas and that I HEARD Katie say things I never would have had we not gone. Absolutely.

I feel in my bones that Neuren checked off all their boxes, too. I think they NEVER would have publically announced their application for Breakthrough Therapy Designation (there’s no FDA rule that says they had to) if they weren’t set for better odds than 30%.

If given all that, the FDA still says no, it just means it’ll take a little longer, that’s all.

But, boy, we’ll be the most “set” parents of anything when that day does come along by then.

I’m all set, now, either way.

Bring on the answer.

This entry was posted in breakthrough therapy, cure, FDA, Neuren Pharmaceuticals, Rett Syndrome,, Trail to a Texas Trial and tagged , , , . Bookmark the permalink.

2 Responses to BTD-2……..Set…… Musing 2

  1. Rosemary Bandura says:

    This feels all too familiar. Kind of like when I was looking for and waiting for a diagnosis for Renee back in the early 1980’s. The frustration of not knowing!

  2. melelllan says:

    Hopefully this wait will be over soon. It will be a relief to have an answer, either way.

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