We’re thrilled to share that a drug involved in the FLAMINGO Auckland study called RCT2100, has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of Cystic Fibrosis (CF). This is a major step forward in the journey to develop new treatments for this challenging genetic condition.
So, what does this mean?
The FDA Orphan Drug Designation is a special status granted to promising treatments for rare diseases like CF. This designation encourages the development of therapies that may not otherwise get the investment or attention they need. Orphan Drug designation can provide incentives such as tax credits for research, support through the regulatory process, and even market exclusivity once approved.
RCT2100, developed by ReCode Therapeutics, is a potential game-changer in the treatment of CF, particularly for people with rare mutations, which are among the more difficult forms of the condition to treat. The drug uses a cutting-edge approach involving messenger RNA (mRNA) and lipid nanoparticles (LNPs) to target the root cause of CF. It’s part of a new wave of science aiming to restore normal function at the cellular level.
New Zealand Clinical Research (NZCR) was directly involved in the Phase 1 clinical trial for RCT2100 right here in Auckland. The study was led by Mayoma Wijesundera, Rohit Katial, and Samantha Nie, with support from our brilliant wider team. Every cohort was delivered in full on time.
A huge congratulations and thank you to everyone involved. This designation brings real hope to families affected by cystic fibrosis around the world. You can read more in ReCode Therapeutics’ official announcement here.
