A New Model for Drug Development in Rare Disease: Gaining Approval as a Class

The CEO of AVI Biopharma is speaking in Geneva tomorrow and the topic of the presentation is very interesting to me, as a father of a boy who is not in the "hot-zone" for current exon-skipping clinical trials.

Gaining approval of AVI or Prosensa treatments as a class is the only way those drugs can help my son in time.

Is anybody at PPMD going attending this conference? If so, can you publish the presentation?


Views: 230

Reply to This

Replies to This Discussion

I'm also very interested in this.  My grandsons don't fall in the "hot-zone" either.

Hello David & Terry!
I checked in with everyone-- Although PPMD will not be attending this meeting, Sharon Terry from Genetic Alliance (USA) will be participating. After the meeting is over, we'll ask around to see if we can get a recap of Chris Garabedian's presentation.


When I asked Pat about this, she also wrote:

Discussions around this topic and efforts are underway to encourage regulatory agencies to approve AON (antisense oligonucleotides) as a class. It is believed that approving the backbone chemistry (prosensa’s  2 Omethyl and/or AVI’s morpholino) would facilitate and expedite approvals of specific sequences. It is estimated, that this approach, could potentially treat 80% +/- of the boys with Duchenne.


This does not mean that the regulatory agencies would step aside, rather they would require a modified toxicology package rather than the full tox package on every chemistry. The difficulty is that each chemistry will express (if successful) a different dystrophin protein and potentially different off target issues.   


In general conversations, regulatory agencies in the US and Europe understand this high unmet need, the need to expedite therapies that are safe and effective. In general conversations both have suggested that once one or two of these AON’s are approved, they would have significantly more data and in a better position to discuss the concept of approving AON as a class.


I hope this helps!


Thank you for passing that on, Stephanie. I am aware it is an ongoing topic of discussion, but mostly from advocacy organizations. I'm interested in how a CEO in the trenches frames the conversation, particularly one who is under intense pressure from investors who have seen AVI stock price degrade significantly this year.

What stresses me out is the statement "once one or two of these AONs is approved...." What if regulators are slow to react and that becomes three, four, five...we don't have that kind of time. I'm hoping to learn that in addition to PPMD and other advocates, the drug companies themselves are putting emphasis on approval as a class now, to maximize the odds that the regulators will react as quickly as possible.

Reply to Discussion


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



© 2022   Created by PPMD.   Powered by

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