top of page
  • Writer's pictureANPDF

Azafaros Media Releases | January 5-9, 2023

"Azafaros Receives FDA's IND Clearance and Fast Track Designation for Lead Asset AZ-3102"

Read the press release from January 5, 2023 here:

20230105_Press Release Azafaros Receives FDA IND Fast Track
Download PDF • 146KB

Read the press release from January 9, 2023 here:

20230109_HR Press Release_final on org changes
Download PDF • 165KB
  • Received Investigational New Drug (IND) clearance from the FDA to conduct a clinical Phase 2 trial for its lead asset, AZ-3102, for the treatment of GM2 and Niemann-Pick disease type C (NP-C).

  • Azafaros’s RAINBOW Phase 2 trial is a multinational, 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 NP-C patients between 12 and 20 years of age.

  • Professor Marc C. Patterson is the principal investigator for the U.S. part of the study.


To join our fight, as well as fund vital research for a cure, support the Australian NPC Disease Foundation in its goals of "Research. Cure. Persevere."

Ways to support NP-C in Australia:

44 views0 comments



bottom of page