Orphan therapies dominate FDA’s September PDUFA dates

FDA’s September review deadlines include at least nine products, six of which are for orphan diseases.

Two of those compounds are for rare pediatric diseases that have no approved treatments per FDA’s Orphan Drug Designations and Approvals database: elamipretide for Barth syndrome and CTX-101 for Menkes disease. While the rare pediatric priority review voucher authorization expired last year, products designated prior to the expiration remain eligible for vouchers if approved before Sept. 30 of next year...