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Hi Paul,
I think they (Prosensa & AVI) suggested waiting until they see any sign of benefit in their systemic trials before the advocacy starts. I just discussed this with Steve Wilton and he seems to be pretty optimistic that only a few compounds need to be tested for this to be approved as a "platform" drug. I do not know what "few" means.
You might be aware that AVI has plans to conduct skipping 51 and 50 trials (51 UK is approved, they are waiting for approvals for 50 USA). Problem is that the 2 trials are not using the same chemistry, skipping 51 will be using PMOs that showed minimal effects on the heart in mice; skipping 50 will be using PPMOs that have great effects on the heart in pre-clinical studies. Since the chemistry is different, these 2 trials will be kept separate in case there are adverse effects with one or the other compound (ie peptide).
As I see it, this will not help speed up the approval process in case one of the compounds gives positive results...
Ofelia
Hi Cheryl,
No timeline until they see any sign of benefit in children in the 51 and/or 50 systemic trials.
At the moment, they have approval from MHRA to conduct the systemic 51 (PMO) UK trail and they are sill waiting for approval from the UK gene therapy committee(I don't recall the exact name) before they can start; the 16 boys who will participate in this trial are already selected.
They are waiting for FDA's approval for systemic skipping 50 (PPMO) USA trial.
Here is an article on Tim Cote.
FDA names orphan products development head
United Press International - September 19, 2007
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WASHINGTON (UPI) -- The U.S. Food and Drug Administration has named Dr. Timothy Cote as director of its Office of Orphan Products Development.
Cote will be responsible for promoting the development of products that demonstrate promise for the diagnosis or treatment of rare diseases or conditions.
A captain in the U.S. Public Health Service Commissioned Corps, Cote most recently served as the Centers for Disease Control's country director for the African nation of Rwanda. In Rwanda, he directed programs in HIV/AIDS, malaria and avian influenza, and was responsible for scientific and administrative leadership in patient care and research initiatives.
Cote -- who succeeds Dr. Debra Lewis, the office's acting director -- previously served as a CDC epidemic intelligence officer at the Maryland Health Department and as chief of the therapeutics and blood safety branch in the FDA's Center for Biologics Evaluation and Research.
The orphan drug program provides a seven-year exclusive right for a pharmaceutical company to market a drug designed to treat a disease that afflicts fewer than 200,000 people in the United States.
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Ofelia,
Thanks for the information. I do get confused by the terminology. I think of ProSensa as the 2'O Methyl company and AviBioPharma as the morpholino company. Is PMO a different acronym for whatever enormous organic molecule ProSensa is using for its exon skipping and PPMO an acronym for AviBioPharma's process?
Ofelia Marin said:Hi Cheryl,
No timeline until they see any sign of benefit in children in the 51 and/or 50 systemic trials.
At the moment, they have approval from MHRA to conduct the systemic 51 (PMO) UK trail and they are sill waiting for approval from the UK gene therapy committee(I don't recall the exact name) before they can start; the 16 boys who will participate in this trial are already selected.
They are waiting for FDA's approval for systemic skipping 50 (PPMO) USA trial.
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