The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to Arcturus Therapeutics Holdings Inc’s product candidate ARCT-032 for the treatment of cystic fibrosis.
ARCT-032 utilizes Arcturus’ Lunar lipid-mediated aerosolized platform to deliver cystic fibrosis transmembrane conductance regulator (CFTR) messenger RNA (mRNA) to the lungs. Expression of a functional copy of the CFTR mRNA in the lungs of people with cystic fibrosis has the potential to restore CFTR activity and mitigate the downstream effects that cause progressive lung disease, according to a release from the company.
The ARCT-032 program is supported by preclinical data in rodents, ferrets, and primates and demonstrates restoration of CFTR expression and function in human bronchial epithelial cells.
The first cystic fibrosis patient in Arcturus’ phase 1b study successfully completed two administrations of ARCT-032, according to the company. Arcturus remains on track to share interim phase 1b data in the first half of 2024.
“Orphan Drug Designation is a very important regulatory milestone in our development plan for ARCT-032,” says Joseph Payne, president and CEO of Arcturus, in a release. “We are executing diligently to accelerate ARCT-032 as a potential new treatment option for people with cystic fibrosis.”
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.
Arcturus is a global late-stage clinical mRNA medicines company focused on the development of infectious disease vaccines and opportunities within liver and respiratory rare diseases.
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