ARCT-032 Gets Orphan Medicinal Product Designation

ARCT-032 Gets Orphan Medicinal Product Designation

By, Admin 26 February 2024

EC grants orphan medicinal product designation to Arcturus Therapeutics’ ARCT-032 to treat cystic fibrosis

Arcturus Therapeutics Holdings Inc., a global late-stage clinical messenger RNA medicines company, announced that the European Commission (EC), based on a positive opinion issued by the European Medicines Agency (EMA), has granted orphan medicinal product designation for the company’s product candidate ARCT-032 to treat cystic fibrosis (CF). In November 2023, the US Food and Drug Administration (FDA) granted orphan drug designation to ARCT-032 for CF.

Orphan Medicinal Product Designation

  • Orphan medicinal product designation by the EC is available to novel therapeutics that prevent or treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 persons in the EU. 
  • The designation provides significant incentives to promote the development of the drug including protocol assistance, access to the centralized authorization procedure, fee reductions, and research grants, as well as 10 years of market exclusivity.

Words from Arcturus Therapeutics

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

Phase I Study

After successful completion of the single-ascending-dose portion of the study in healthy adults, the first participant with CF in the phase 1b study part enrolled and completed two administrations of ARCT-032. Arcturus remains on track to share interim phase 1b data in H1 2024.

Cystic Fibrosis

  • Cystic fibrosis is a life-shortening disease with a worldwide distribution. Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene result in a reduction or absence of CFTR protein and/or function in the airways, causing insufficient chloride transport to maintain airway surface homeostasis. 
  • CF mucus is more difficult to clear, thus clogging the airways and leading to infection, inflammation, respiratory failure, or other life-threatening complications. 
  • Currently approved CFTR modulator therapies are designed to increase function of the CFTR channel to help reduce symptoms yet are ineffective in some people with CF because of their underlying mutations.

About ARCT-032

  • ARCT-032 utilizes Arcturus' LUNAR lipid-mediated aerosolized platform to deliver CFTR messenger RNA to the lungs. 
  • Expression of a functional copy of the CFTR mRNA in the lungs of people with CF has the potential to restore CFTR activity and mitigate the downstream effects that cause progressive lung disease. 
  • The ARCT-032 programme is supported by preclinical data in rodents, ferrets and primates, as well as demonstrating restoration of CFTR expression and function in human bronchial epithelial cells.

About Arcturus Therapeutics

Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. is a global late-stage clinical mRNA medicines and vaccines company with enabling technologies: (i) LUNAR lipid-mediated delivery, (ii) STARR mRNA Technology (sa-mRNA) and (iii) mRNA drug substance along with drug product manufacturing expertise.

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