
Glafabra is evaluating consultant support for our regulatory submissions, and Tim Cote presents a compelling option due to his affordability and highly relevant experience. In his 24 year tenure at the FDA, became Director of Office of Orphan Products Development (OOPD) from 2007 to 2011. Following his FDA service, Tim became Chief Medical Officer for National Organization for Rare Disorders (NORD). He then established a successful consulting firm in 2012, which IQVIA acquired in 2017 for $20 million.. Next, Tim formed Only Orphans Cote allowing him to renew his consulting efforts strictly to rare disease targets.
Glafabra needs to get Orphan Drug Designation (ODD) with the FDA as quickly as possible. Tim’s costing estimates are $65,000 to get Glafabra to an ODD. Tim estimates the average chance of first time success is near 40%. However, he says the success rate for projects he consults on is near 95%. An ODD for our Fabry asset provides a significant amount of de-risking for our therapy approach. Having an ODD will make Glafabra become a more attractive candidate for investing and partnering.
At the same time, we will start efforts to get an open IND. This will require we have either an Interact meeting or a PreIND meeting with the FDA. Tim say we can request one type, but the ultimate meeting type will be at the discretion of the FDA. Tim’s team estimates the cost to execute an IND discussion with his team’s guidance to be at $95K. Tim recommends we also focus on motivating patient groups to be involved, so we will continue to cultivate these types of relationships with patient advocacy groups.
Bottomline, the costing estimate to get to preIND meeting achieved status under an orphan drug designation is near $155,000. Accordingly we have now added this as a line item in the use-of-proceeds for our preseed raise.