Dicerna’s DCR-PHXC a Breakthrough Therapy for rare kidney disorder

The FDA has designated Dicerna Pharmaceuticals’ (DRNA +0.5%) RNAi candidate DCR-PHXC a Breakthrough Therapy for the treatment of a rare inherited kidney disorder called primary hyperoxaluria type 1 (PH1).

PH2 and PH3 may be added in the future.

A Phase 2 study, PHYOX2, in PH1 and PH2 is recruiting patients.

Breakthrough Therapy status provides for more intensive guidance from the FDA on development, the involvement of more senior agency personnel and a rolling review of the marketing application.

https://seekingalpha.com/news/3477771-dicernas-dcr-phxc-breakthrough-therapy-rare-kidney-disorder