Historically, drug development within the rare disease space has been stifled; companies tend to focus on developing therapies for bigger patient populations that will be more impactful and more profitable. The Orphan Drug Act was designed to incentivize rare disease drug development. Now, Orphan Drug designation, which is granted to therapies for conditions affecting fewer than 200,000 people nationwide, comes with benefits such as fee waivers, increased regulatory assistance, and seven years of market exclusivity if/when the drug is approved. According to reporting by Healio, the U.S. Food and Drug Administration recently granted Orphan Drug designation to ARCT-032 for cystic fibrosis (CF).

ARCT-032 is an inhalable cystic fibrosis transmembrane conductance regulator (CFTR) mRNA replacement therapy. This antifibrotic treatment is delivered straight to the lungs using Arcturus Therapeutics’ proprietary LUNAR aerosolized platform.

Data on ARCT-032 from a Phase 1b clinical study should be available earlier in next year.

Understanding Cystic Fibrosis

The CFTR gene normally helps regulate salt movement throughout the body. People with cystic fibrosis, a progressive genetic disorder, have CFTR mutations that affect this movement. Because of this, people with cystic fibrosis have thicky, sticky mucus that accumulates in their respiratory and digestive systems – as opposed to healthy, more “slippery” mucus. This thick mucus can stop digestive enzymes from being released and make it harder to breathe. Cystic fibrosis is more common in Caucasian newborns. It once came with a poor prognosis. However, treatments have significantly improved overall lifespan. Available treatments include Kalydeco, Orkambi, Symdeko, Trikafta, and a variety of other medications, pulmonary rehabilitation, and surgical interventions.

People with cystic fibrosis may have symptoms such as:

• A persistent cough that produces thick mucus
• Frequent or recurrent respiratory infections
• Difficulty exercising / exercise intolerance
• Shortness of breath
• Wheezing
• Recurrent sinusitis
• Nasal polyps
• Clubbing of the fingers and toes
• Intestinal blockages in newborns
• Salty-tasting skin
• Greasy, foul-smelling stools
• Chronic or severe constipation that could lead to rectal prolapse
• Male infertility
• Pancreatitis (more common in people not diagnosed until adulthood)