Posted by Michael Wonder on 06 Jan 2023
Azafaros receives fast track designation for lead asset AZ-3102
5 January 2023 - Azafaros today announced that the FDA has granted fast track designation for the investigation of AZ-3102 for GM1 and GM2 gangliosidoses and Niemann-Pick disease type C.
Azafaros’ RAINBOW Phase 2 trial is a multi-national, double-blind, placebo-controlled, parallel-group trial investigating the safety, tolerability, and pharmacokinetics of a once daily oral administration of AZ-3102 for 12 weeks in GM2 and Niemann-Pick disease type C patients between 12 and 20 years of age.
