Challenge Accepted!

From: Trail to a Texas Trial

Challenge Accepted!

It hasn’t happened before. I was given a MISSION, if I chose to accept it, to explain some new developments in regards to Sarizotan (Newron) and Anavex 2-73 (Anavex) receiving FDA Rare Pediatric Disease Designation: “Mel Lancaster have you posted about these announcements or am I just behind on things? Have you deciphered it for parents who are new and may not get all of this yet?” Let me just say I was quite flattered! So, never one to step away from a challenge… Here we go.

One of the first hurdles for these companies and drugs to get past is Orphan Drug Designation. “The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the US or meets cost recovery provisions of the act.” (From the FDA website)

A “Rare Pediatric Disease”, in contrast, is a rare disease “and one where the disease is serious or life-threatening with the serious or life-threatening manifestations primarily affecting individuals from age zero to 18.” (From the FDA website). Also, if a rare disease begins in childhood but doesn’t become life threatening until adulthood, it doesn’t qualify. If a disease is rare, begins in childhood, but isn’t life-threatening, it also does not qualify.

So, this is a subtle difference, but important. Now, Orphan Drug status and Rare Pediatric Disease may seem synonymous to a degree, however they are not, nor are the incentives for the companies. And this difference in incentives is the BIGGEST reason this new designation for these companies is so important.

In order to understand what is such a big deal about this, one must have a basic understanding of the different designation/statuses that these drugs/companies have or may already have. And before anyone asks, yes Trofinetide/Neuren has most if not all of the following.

From the FDA website:

Fast Track status: Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need.

  • More frequent meetings with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval
  • More frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers
  • Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met
  • Rolling Review, which means that a drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. BLA or NDA review usually does not begin until the drug company has submitted the entire application to the FDA

Priority Review: A Priority Review designation means FDA’s goal is to take action on an application within 6 months. (instead of 10 months) *This is the BIG DEAL about Rare Disease Designation*

Orphan Drug Status: An orphan drug is a drug meant for a rare disease or condition. With this designation, the company receives incentives to continue to develop this drug. Some of the incentives include:

• 7-year marketing exclusivity

• Tax credits (up to 50% of clinical development costs)

• Exemption/Waiver of application (filing) fees

• OOPD assistance during the development process

Rare Pediatric Disease Designation: “The Rare Pediatric Disease designation provides incentives to advance the development of rare disease drugs and biologics. Additionally, the FDA’s Rare Pediatric Disease Priority Review Voucher Program states that a sponsor with a Rare Pediatric Disease designation who receives marketing approval for a rare pediatric disease may be eligible for a voucher that can be redeemed to obtain priority review for any subsequent marketing application.” (From Business Wire)

And, therein lies the biggest part of this news. Having drugs be given Rare Pediatric Disease Designation makes it POSSIBLE to apply for the priority review. A priority review means the FDA must take action on a MARKETING application within 6 months instead of 10.  IF a company already received a priority review through another process (i.e. Fast Track Status- which is then specific for THAT drug only), this is theirs to do, in particular, one of two things. 1. They can keep it and use it for another drug, even if it is not rare or even for a pediatric disease, OR 2. they can sell it for tens of MILLIONS of dollars and more. If they DON’T have it already and get it through this program, then they can use it to speed up the time from the application submission to when they can begin to market the drug.

Priority Review is important to US, because it gets a drug, if approved, to our children approximately 4 months faster (give or take). But, if a drug already has priority review and then it gets ANOTHER through the Rare Pediatric Disease (RPD) Designation Program, it’s theirs to keep or sell. For the company/ies this is a tremendous monetary advantage .

I congratulate both Newron and Anavex on this accomplishment.

Challenge Accepted!

A Day with ACADIA- Part 2: Assurance

Some of the ACADIA employees at the Rett syndrome Awareness event.

Katelin and I had the great pleasure of being invited to ACADIA a couple of weeks ago for Rett syndrome Awareness Month. Last week I posted Part 1: Awareness; I had PLANNED to follow that closely with Part 2, but a little thing called the “Official initiation of Phase III” interrupted that plan! But, this part is especially important now that the trial has officially started.

I have held ACADIA and Bill Keller, Executive Director; Patient Advocacy and Industry Relations, in high esteem since I first met them over a year ago. I was struck by their commitment to the families- how can we make this process easier for families. I was reassured by how important they believed US to be in this process. They interviewed multiple families, had an in-person roundtable with families in Princeton, and reached out to people and organizations that could give insight as to how to best run the trial in a way that supported families. I was reassured then, now I was given assurance.

One thing Bill Keller said that really resonated with me because of the sincerity and sense of duty that he said it with- (paraphrasing here) “I met Steve Kaminsky and he told me that since this drug (Trofinetide) existed it was our responsibility to get it to families.”

And that sense of responsibility is clear in everything ACADIA does-from listening to their employees’ desire to learn more about Rett syndrome from families to setting up a website weeks ago that put families in touch with sites. Despite all that, the one thing that sealed the deal for me was this-

ACADIA wants to hear from US, the Rett community-

  • Can you please let me know, so that I can share with ACADIA, the resources and support that you value most in caring for your Rett child by way of getting new information?
  • We also talked about community support programs such as Strollathons etc. but it would be great to know, if ACADIA attended these events, what information would you value the most. Keeping in mind they aren’t able to divulge anything about how the trial is going, but should they be a presence at these events ACADIA wants to make sure that they are providing something of value and not information we already know, which would cause frustration.

Let’s pull together and give them options to choose from. This is not a company that is saying… we’re the scientists, you’re just going to have to wait until it’s all over and deal with it… they’re saying, “We are in this together.” Something along the lines of “It takes a village” to do this Phase III and all its aspects right.

What assurances did we get?:

That they are systematically having sites inspected and rolling them out as soon as possible.

That all issues with manufacturing-such as multidose storage etc. have been resolved.

The mere fact that the trial is a go means they have enough Trofinetide in four different tiered doses for every child in the trial and for Lilac 2-an extension to (hopefully) cover from the end of the trial to approval.

That ACADIA has a plan in place to assist families with the costs of trial participation.

That ALL the families matter, those in the trial and those that don’t qualify because we’re all going to get it if (when) it gets approved.

I will forward suggestions to ACADIA in a couple of weeks, giving YOU-parents/grandparents/caregivers a chance to participate in a real way. ACADIA feels that trial participants are not the only ones who can make a difference. Here is a chance for those with children/adults that don’t qualify for the trial itself to contribute in a positive and meaningful way.

You can leave a comment here, on my main blog: Trail to a Texas Trial, on Facebook or email me at m3ml@hotmail.com. 

We’ve got this. We’re in good hands with ACADIA and Neuren.

A Day with ACADIA- Part 2: Assurance

Hot off the Presses- The Horses are Outta the Gate!

Our jockey wears a purple hat!

Well yesterday’s post WAS going to be the last one this month but then I got an email from Acadia- “Hot off the Presses” it said and then this announcement- ACADIA Pharmaceuticals Initiates Phase 3 Pivotal Study of Trofinetide in Rett Syndrome, a Rare Neurodevelopmental Congenital CNS Disorder  The tears started around the word “initiates” and by the time I got to “Trofinetide” I was a blubbery mess, snot and all. Within minutes of posting it to Facebook, I got a call from another mom and there we were the two of us crying, swearing, cheering, making no sense and so much sense all at the same time. I count that as one of the precious memories I hope to carry all the way to my grave.

Following quickly on the heels of that was this announcement from Acadia- Trofinetide Phase-3 Clinical Study Update There’s a lot of pertinent information in there, so please take a look. The thing that jumped out at me are three sites- Phoenix, AZ; Houston, TX; Chicago, IL- have already started screening. Now, I KNOW some parents are getting responses along the lines of… we already have so many interested families… do not be discouraged, reach out to sites further down the line and also remember unfortunately not every child screened will pass the process, so we NEED more interested families than spots in order for them all to get filled.

And lest we forget where it all began- Neuren released its own announcement . So, in case you all couldn’t guess, this is BIG, BIG NEWS.

I know that as we each have read these announcements the possibilities that come to us are all different. In a moment the “never gonna’s” have become “maybe will’s”, “could it really be possible’s” and while I am totally on the side of Trofinetide making a difference, I can’t possibly guess if all girls will be affected eventually in the same way, but I believe with all my heart and soul that at the very least some of your “never gonna’s” are going to go away. I believe you will hear the lilt of your child’s voice in some way that everyone will hear and understand. I believe hands will do things that may be very small or huge and it will seem like a miracle.

There are still things we need to know- is there a cap on improvement, meaning eventually is there a place where our children can’t improve anymore when we see there is still room? Are there side effects with long term use? Each and every family that partakes are heroes to all of us; even those who don’t end up qualifying, you tried and that’s everything in a world where trying can be so hard and defeating, it’s so very brave to take that chance.

The race has begun, the horses are out of the gate; I’m betting on ours, the one with the purple-headed jockey; I’m betting on us.

Hot off the Presses- The Horses are Outta the Gate!