Arcturus Therapeutics Has Been Granted Orphan Drug Designation by the U.S. FDA for ARCT-032, Intended for the Treatment of Cystic Fibrosis

28 November 2023

Arcturus Therapeutics Holdings Inc. (NASDAQ: ARCT) has recently announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for ARCT-032, the company's product candidate for treating cystic fibrosis (CF). This regulatory milestone is a significant step in Arcturus' development plan for ARCT-032, which aims to provide a new treatment option for individuals with CF.

Orphan Drug Designation is awarded by the FDA's Office of Orphan Products Development to drugs designed for rare diseases affecting fewer than 200,000 people in the United States. This designation offers substantial incentives, including potential market exclusivity for seven years upon FDA approval, eligibility for tax credits related to qualified clinical trials, a waiver of the Prescription Drug User Fee Act Application fee, and access to regulatory guidance from the FDA for the overall drug development plan.

Arcturus Therapeutics, emphasized the importance of this regulatory milestone and the company's commitment to advancing ARCT-032 as a potential treatment option for cystic fibrosis.

The Phase 1b study of ARCT-032 has successfully administered two doses to the first CF patient, and interim Phase 1b data are expected to be released in the first half of 2024.

Cystic fibrosis is a globally prevalent, life-shortening disease resulting from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, leading to a lack or reduction of CFTR protein and/or function in the airways. ARCT-032 utilizes Arcturus' LUNAR® lipid-mediated aerosolized platform to deliver CFTR messenger RNA to the lungs. This approach has shown promise in preclinical studies, with evidence of restored CFTR expression and function in various animal models, as well as in human bronchial epithelial cells.