In human bronchial epithelial cells, ARCT-032 was shown to restore expression and functioning of CFTR. Credit: Marko Aliaksandr / Shutterstock.com.

The US Food and Drug Administration (FDA) has granted orphan drug designation for Arcturus Therapeutics’s ARCT-032 for the treatment of cystic fibrosis (CF).

The product candidate leverages the company’s LUNAR lipid-mediated aerosolised platform to deliver cystic fibrosis transmembrane conductance regulator (CFTR) messenger RNA (mRNA) to the lungs. 

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The expression of a functional copy of CFTR mRNA in the lungs could aid in restoring CFTR activity in CF patients, reducing disease progression. 

The ARCT-032 programme is backed by preclinical findings in ferrets, rodents and primates.

It restored the expression and functioning of CFTR in human bronchial epithelial cells.

In the Phase Ib clinical trial, the first patient with CF received two doses of ARCT-032. 

The company plans to report interim data from the trial in the first half of 2024.

Arcturus Therapeutics president and CEO Joseph Payne stated: “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.”

The orphan status has been granted by the FDA’s Office of Orphan Products Development for drugs in the development stage and for the prevention of rare diseases that impact fewer than 200,000 individuals in the US. 

It incentivises drug development with market exclusivity for seven years following approval, tax credit eligibility for clinical trials and regulatory guidance.

In September 2023, the company extended an agreement with the Cystic Fibrosis Foundation to advance the development of ARCT-032.