The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for Arcturus Therapeutics’ product candidate ARCT-032 to treat cystic fibrosis (CF).
The FDA’s Office of Orphan Products Development grants orphan status to drugs being developed to treat, prevent, or diagnose a rare disease or condition affecting fewer than 200,000 people in the United States.
The designation provides significant incentives to promote the development of the drug including the potential for market exclusivity for seven years upon FDA approval, eligibility for tax credits for qualified clinical trials, waiver of Prescription Drug User Fee Act Application fee, and eligibility to receive regulatory guidance from the FDA in the design of an overall drug development plan.
“Orphan Drug Designation is a very important regulatory milestone in our development plan for ARCT-032,” said Joseph Payne, President, and Chief Executive Officer of Arcturus Therapeutics. “We are executing diligently to accelerate ARCT-032 as a potential new treatment option for people with cystic fibrosis.”
The first CF patient in Arcturus Therapeutics’ Phase 1b study successfully completed two administrations of ARCT-032. Arcturus Therapeutics, a global late-stage clinical messenger RNA medicines company focused on the development of infectious disease vaccines and opportunities within liver and respiratory rare diseases, remain on track to share interim Phase 1b data in H1 2024.