Arcturus Therapeutics gains FDA ODD for cystic fibrosis treatment
Betsy Goodfellow | November 28, 2023 | News story | Research and Development | Arcturus Therapeutics, Chronic Diseases, FDA, ODD, cystic fibrosis
Arcturus Therapeutics has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ARCT-032, the company’s cystic fibrosis (CF) treatment.
The company has dosed one CF patient in a phase 1b study, who has completed two administration of the drug. Interim data is expected to be shared early in 2024.
The FDA’s Office of Orphan Products Development grants ODD to drugs in development for the treatment, prevention or diagnosis of rare diseases of conditions which affect fewer than 200,000 people in the US.
ACRT-032 utilises the company’s Lunar lipid-mediated aerosolized platform in order to deliver CFTR messenger RNA to the lungs. The programme is supported by preclinical data in rodents, ferrets and primates.
Joseph Payne, president and chief executive officer of Arcturus Therapeutics, commented: “Orphan Drug Designation is a very important regulatory milestone in our development plan for ARCT-032. We are executing diligently to accelerate ARCT-032 as a potential new treatment option for people with cystic fibrosis.”
Betsy Goodfellow
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