At the NBIA Family Meeting in May and in our follow-up communication to CoA-Z study participants, we mentioned the possibility of our doing one or more short follow-up studies of CoA-Z to supplement our clinical trial data. After further data analysis and reflection, we have decided that our current data is strong enough to submit to the FDA without doing any additional clinical studies. If the FDA agrees, then this will be the fastest path to receiving the approvals we need to make the compound more widely available to the PKAN community.

So we are setting aside any plans for follow-up studies for now, instead directing all our efforts to compiling our data to present to FDA later this fall. It is a big task, because it not only involves data from the CoA-Z clinical trial, but also from all the related background work going back to 2015: more than 500 pages in length! We don’t have the resources to offer further access to CoA-Z before FDA approval, and so we feel this is the best path to make it broadly available to the PKAN community.

The studies we were considering would have posed quite a burden on participants (such as several blood draws over a short time period) and the studies would not have been long enough to offer any real expectation of benefit, so we hope the PKAN community will see this as good news – we do! We know the community is deeply interested in our progress, and we will continue to update you.

– The OHSU CoA-Z trial team