Policy & Regulation
Arcturus Therapeutics granted FDA Orphan Drug Designation for ARCT-032 to treat cystic fibrosis
27 November 2023 -

Late-stage clinical mRNA medicines company Arcturus Therapeutics Holdings Inc (Nasdaq: ARCT) announced on Monday that it has received Orphan Drug Designation from the U.S. FDA for ARCT-032 in cystic fibrosis (CF) treatment.

The designation offers incentives, including potential market exclusivity for seven years upon FDA approval. The Phase 1b study's first CF patient completed two administrations of ARCT-032, with interim Phase 1b data expected in H1 2024.

CF is a globally distributed life-shortening disease caused by mutations in the CFTR gene. ARCT-032 utilises Arcturus' LUNAR lipid-mediated aerosolised platform to deliver CFTR messenger RNA to the lungs, potentially restoring CFTR activity.

Arcturus was founded in 2013 and is based in San Diego. It has various enabling technologies, including LUNAR lipid-mediated delivery and STARR mRNA Technology. The pipeline includes candidates for ornithine transcarbamylase deficiency and cystic fibrosis, along with mRNA vaccine programs for COVID-19 and influenza.