ARCT-032 granted orphan drug status in European Union

Experimental therapy previously received similar designation in US

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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The European Commission has granted orphan drug designation to ARCT-032, an inhaled experimental treatment for cystic fibrosis (CF) developed by Arcturus Therapeutics.

Orphan drug designation is designed to encourage developing therapies for rare and serious diseases, those affecting not more than 5 in every 10,000 people in the European Union (EU). It includes benefits such as 10-year market exclusivity in the EU upon approval, reduced fees, and access to the EU’s centralized marketing authorization procedure.

The decision, based on the favorable opinion of the Committee for Orphan Medicinal Products, follows a similar designation by the U.S. Food and Drug Administration last year. The therapy candidate also has received rare pediatric disease designation in the U.S., which is designed to boost the development of medicines for conditions mainly affecting children.

“We are pleased to receive orphan medicinal product designation from the EC [European Commission] for ARCT-032, as it represents a significant milestone for our CF program,” Joseph Payne, president and CEO of Arcturus, said in a company press release. “We will continue to advance ARCT-032 as a potential new treatment option for people with CF regardless of their genetic mutations.”

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Messenger RNA molecule part of ARCT-032

CF is caused by mutations in the CFTR gene, which lead to or impaired production of a protein of the same name. This results in the accumulation of a thick and sticky mucus in the lungs and other organs.

ARCT-032 contains the messenger RNA molecule — a template for protein production derived from DNA — that has the information for making the CFTR protein. This means that ARCT-032 may treat all CF patients regardless of their mutation, according to the company, unlike CFTR modulators that are ineffective in some people with CF because of their underlying mutations.

Designed using their LUNAR platform, the therapy is delivered by inhalation of tiny, fat-like particles that deliver their cargo to lung cells, allowing a functional CFTR protein to be produced.

A Phase 1a clinical study (NCT05712538) testing the safety and tolerability of single ascending doses ARCT-032 in healthy adults has been completed. A subsequent Phase 1b study is underway to test ARCT-032 in up to eight adults with CF. A first patient has received two doses of ARCT-032 two days apart.

Its goal is to assess ARCT-032’s safety, tolerability, and pharmacokinetics, or its movement into, through, and out of the body. The impact on lung function and mucus clearance also will be assessed as exploratory measures. Follow-up assessments will be made over a four-week period.

According to the company, interim data are expected in the first half of this year.