FDA states Exon cocktail must go through clinical trials for each exon!

I read that FDA is putting a hold on exon skipping. FDA is now requiring that each exon goes through clinical trial. We were hoping beyond hope that all exons would be approved without each exon formulation having to go through the clinical trial. If you are unfamiliar with the clinical trial process, then google it and you will see it takes years and tons of money - money that the drug company itself has to pay for. It's not even about the money right now - it is about the time factor....

Our son has a duplication of 54-57. If DMD is rare and duplications are only 20% of that - then exon trails for duplication of our son's = being so far out in time, if ever at all. How does a parent retain hope? We raise awareness, donate money, research and our life becomes finding a cure....And, now when one is so close, the government steps in and puts on hold on it??? Life is not fair.....And for those boys that do have exon 50 or 51 - they get "the cure" down the line? Tell me - where is faith and hope???

Even a parent who emails and calls the FDA's chief person and can't get compassionate use approved....
Sorry to be negative - but this is one of the few places that parents understand my writings.....
Char Burke
son of Will - age 6 - duplications 54-57

Views: 750

Reply to This

Replies to This Discussion

I have looked into this in some detail, both in reading the law myself and in consultation with others who have direct experience in advocating before the FDA. The FDA has the legal right to require its full process for each separate PMO, PPMO or 2OM (the Prosensa chemistry) exon skipping compound; they also have the discretion to require less than that. Last year, Tim Cote made it pretty clear that his office, at least, was going to have an open mind on this, but his office doesn't have the last word, and I think that even they would need to have good, evidence based, reasons for believing that demonstration of safety and efficacy for one compound will be similar for similar compounds targeting different exons. AviBioPharma appears to believe, based on statements made last September, that the European regulator is expecting to not require testing for each compound, and may only require the full sequence of trials for one compound in each "family." It's a long grind in any event; this is simply one bet to be covered with many other compounds showing as much promise and not much further behind in the pipeline. In the case of Losartan and Idebennone, the end of the pipeline is simply closer if adequate funding can be found to test drugs which aren't patent protected and can't be portrayed as keys to the locks separating yuppies from immortality like exon skipping's promoters like to pretend when they're stumping for venture capital.
To maximize the chances of FDA approving exon skipping as a posiible treatment for ALL the exons, AVI should consider trials using variety of exons. I hope someone considers this rather than just betting on FDA to approve all after looking at 1 successful exon skipping trial.
From what I recall of the September investor's presentation that you can still listen to at their website, this is exacly what they intend as soon as they sign up a partner with deep enough pockets to run the multiple trials simultaneously. They claim to have four additional compounds teed up.

http://www.avibio.com/pr/pr407.php

Ofelia,

you listened to the January 21, 2009 presentation, did they identify exons for which they have compounds formulated?

Bains said:
To maximize the chances of FDA approving exon skipping as a posiible treatment for ALL the exons, AVI should consider trials using variety of exons. I hope someone considers this rather than just betting on FDA to approve all after looking at 1 successful exon skipping trial.
About FDA's clinical hold on AVI-4658, I had some time to look again at Leslie Hudson's presentation from September 08:

http://www.avibio.com/pr/pr390.php

http://www.avibio.com/downloads/AVI-2008-09-10-03-Leslie_Hudson_DMD...

On slide 16 he has "US plans for Clinical Development of AVI-4658".

-Negotiated preclinical requirements with the FDA
-Have allowance for mechanistic toxicology study in mdxmouse model of DMD to support clinical testing of AVI-4658
–Evaluation of PMO-based exon 23 drug
Initiate 3-month mdxtoxicology study in 4Q 2008
-Anticipate that UK Phase 1b study results might impact FDA decision making prior to completion of planned pre-clinical GLP agenda for US-based studies
–Might allow immediate pivotal trial testing in US DMD boys


From this it is very clear that their preclinical work is not completed, which explains the clinical hold.




Paul Cliff said:
From what I recall of the September investor's presentation that you can still listen to at their website, this is exacly what they intend as soon as they sign up a partner with deep enough pockets to run the multiple trials simultaneously. They claim to have four additional compounds teed up.

http://www.avibio.com/pr/pr407.php

Ofelia,

you listened to the January 21, 2009 presentation, did they identify exons for which they have compounds formulated?

Bains said:
To maximize the chances of FDA approving exon skipping as a posiible treatment for ALL the exons, AVI should consider trials using variety of exons. I hope someone considers this rather than just betting on FDA to approve all after looking at 1 successful exon skipping trial.
There not going be many investors with deep enough pockets to invest in something what converts DMD to BMD with unknown output in most of cases. I am not even sure how many investors would have enough patientce to wait for the aproval of the drug with multiple trials guided by FDA.Before most of exons would be tested , I would bring my son in the urn for some another meeting ......in ..2025.
Let's hope that won't be the case. I think think the expectation that even postponing scoliosis surgery, invasive ventilation, 24 hour nursing and and the other expensive medical interventions our kids are going to need in the second halves of their lives is going to be of tremendous financial benefit to the insurers or public health agencies. Thus, the drug companies, at least Avi BioPharma, expects to be able to charge many tens of thousands per year. Even with our small patient population, these dollar amounts might move the needle and could justify running trials simultaneously instead of consecutively. There is no doubt that other approaches need to be pursued at the same time.


BOZ4J said:
There not going be many investors with deep enough pockets to invest in something what converts DMD to BMD with unknown output in most of cases. I am not even sure how many investors would have enough patientce to wait for the aproval of the drug with multiple trials guided by FDA.Before most of exons would be tested , I would bring my son in the urn for some another meeting ......in ..2025.

Dear Bains,

I have two boys 5and half and 13 months, for both, the exon 11 must be skipped for obtaining a readable code. Kindly let me if you have found any answers yet. Thanks and kind regards

Joseph

Bains said:

I HOPE this is not the case. My son needs exon 11 skipping and god knows when will they be able to do that.

Dear Bains,

I hope that you and your child are fine by grace of God. I have two children 9 and 5 years old, we too am looking to find if there is any advancement in Exon 11 skipping yet.

Please let me know if you hear any thing

God bless and best regards

Vinu Joseph

Qatar

Bains said:

I HOPE this is not the case. My son needs exon 11 skipping and god knows when will they be able to do that.

Reply to Discussion

RSS

Need help using this community site? Visit Ning's Help Page.

Members

Events

© 2021   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service