Arcturus Therapeutics Gets FDA Orphan Designation for ARCT-032 in Cystic Fibrosis

By Colin Kellaher

Arcturus Therapeutics Holdings has won U.S. Food and Drug Administration orphan-drug designation for its ARCT-032 product candidate to treat the life-shortening genetic disease cystic fibrosis.

The San Diego late-stage clinical messenger RNA medicine company on Monday said the first patient in its Phase 1b study has successfully completed two administrations of ARCT-032, adding that it is on track to share interim data from the study in the first half of 2024.

The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S. and provides for an extended marketing exclusivity period against competition.

Write to Colin Kellaher at [e-mail]

(END) Dow Jones Newswires

November 27, 2023 08:58 ET (13:58 GMT)

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