Arcturus Granted Orphan Designation by European Commission in Win for Cystic Fibrosis Patients
Arcturus Therapeutics (Nasdaq: ARCT) has been granted orphan medicinal product designation by the European Commission (EC) for ARCT-032 in the treatment of Cystic Fibrosis (CF). The company received Orphan Drug Designation from the FDA for ARCT-032 in November 2023.
Joseph Payne, President & CEO of Arcturus Therapeutics, said, “We are pleased to receive orphan medicinal product designation from the EC for ARCT-032, as it represents a significant milestone for our CF program. We will continue to advance ARCT-032 as a potential new treatment option for people with CF regardless of their genetic mutations.”
Arcturus expects to share interim Phase 1b data in H1 2024.
Highlights
ARCT-032 utilizes Arcturus’ LUNAR® lipid-mediated aerosolized platform to deliver CFTR messenger RNA to the lungs.
After successful completion of the single-ascending-dose portion of Arcturus’ study in healthy adults, the Phase 1b study has commenced. The first participant with CF has been enrolled and has completed two administrations of ARCT-032.
Cystic fibrosis is a life-shortening disease characterized by infection, inflammation, respiratory failure, or other life-threatening complications.
About Arcturus Therapeutics
Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT) is a global late-stage clinical mRNA medicines and vaccines company with enabling technologies: (i) LUNAR® lipid-mediated delivery, (ii) STARR® mRNA Technology (sa-mRNA) and (iii) mRNA drug substance along with drug product manufacturing expertise. Arcturus developed the first self-amplifying messenger RNA (sa-mRNA) COVID vaccine in the world to be approved. Arcturus has an ongoing global collaboration for innovative mRNA vaccines with CSL Seqirus, and a joint venture in Japan, ARCALIS, focused on the manufacture of mRNA vaccines and therapeutics. Arcturus’ pipeline includes RNA therapeutic candidates to potentially treat ornithine transcarbamylase deficiency and cystic fibrosis, along with its partnered mRNA vaccine programs for SARS-CoV-2 (COVID-19) and influenza. Arcturus’ versatile RNA therapeutics platforms can be applied toward multiple types of nucleic acid medicines including messenger RNA, small interfering RNA, circular RNA, antisense RNA, self-amplifying RNA, DNA, and gene editing therapeutics. Arcturus’ technologies are covered by its extensive patent portfolio (over 400 patents and patent applications issued in the U.S., Europe, Japan, China, and other countries). For more information, visit www.ArcturusRx.com. In addition, please connect with us on Twitter and LinkedIn.
