FDA grants orphan drug designation to cystic fibrosis treatment

The FDA granted orphan drug designation to ARCT-032 as a treatment for patients with cystic fibrosis, according to a press release from Arcturus Therapeutics.

Underlying mutations in patients with cystic fibrosis can impact the efficacy of the cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies that are currently approved, according to the release.

ARCT-032 (Arcturus Therapeutics) delivers CFTR messenger RNA to patients’ lungs and is capable of restoring CFTR expression and function in human bronchial epithelial cells.

The release highlights that the drug conducts this delivery through the company’s LUNAR lipid-mediated aerosolized platform.

As indicated by the release, Arcturus Therapeutics plans to share interim phase 1b data in the first half of 2024. The company also noted the first patient in the trial has successfully received two administrations of the drug.

“Orphan drug designation is a very important regulatory milestone in our development plan for ARCT-032,” Joseph E. Payne, president and CEO of Arcturus Therapeutics, said in the release. “We are executing diligently to accelerate ARCT-032 as a potential new treatment option for people with cystic fibrosis.”

Orphan drug designation is given to treatments that address rare diseases affecting fewer than 200,000 people in the United States. The designation provides assistance in developing drugs, tax credits, exemptions from FDA fees and 7 years of marketing exclusivity.