Press release
Arcturus Therapeutics Receives Orphan Drug Designation from the U.S. FDA for ARCT-032, for the Treatment of Cystic Fibrosis
First cystic fibrosis patient in Phase 1b study successfully completed two administrations of ARCT-032 On track to share interim Phase 1b data in H1 2024 SAN
About this update from Arcturus Therapeutics Holdings Inc.
[{"type":"text","content":"\nFirst cystic fibrosis patient in Phase 1b study successfully completed two administrations of ARCT-032\n\n\nOn track to share interim Phase 1b data in H1 2024\n\n\n SAN DIEGO--(BUSINESS WIRE)--\nArcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), 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, today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the Company’s product candidate ARCT-032 to treat cystic fibrosis (CF).\n\n\nThe 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.\n\n\n“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.”\n\n\nThe first CF patient in our Phase 1b study successfully completed two administrations of ARCT-032. We remain on track to share interim Phase 1b data in H1 2024.\n\n\nAbout Cystic Fibrosis\n\n\nCystic fibrosis is a life-shortening disease with a worldwide distribution. Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene result in a reduction or absence of CFTR protein and/or function in the airways, causing insufficient chloride transport to maintain airway surface homeostasis. CF mucus is more difficult to clear, thus clogging the airways and leading to infection, inflammation, respiratory failure, or other life-threatening complications. Currently approved CFTR modulator therapies are designed to increase function of the CFTR channel to help reduce symptoms yet are ...