Dec. 02, 2019: Akari Therapeutics announced the design of a pivotal Phase III pediatric trial in HSCT-TMA following a FDA End-of-Phase II meeting.
Akari announced that in another hematological condition, the patients after  long-term Phase II PNH study are now became transfusion independent on long-term treatment with nomacopan, who were  transfusion dependent.
HSCT-TMA is an orphan hematological condition with an estimated mortality rate of more than 80% in children with the severe form of the disease and is  being targeted with nomacopan. The study will be in two parts, Part A with data from seven patients used to confirm dosing and endpoints for Part B, with the pharmacokinetic (PK) modelling approved with the FDA through Akari’s participation in the Model Informed Drug Discovery Program (MIDD). https://fda.einnews.com/pr_news/503778385/akari-therapeutics-announces-pivotal-phase-iii-trial-design-of-nomacopan-in-pediatric-hematopoietic-stem-cell-transplant-related-thrombotic
