Novel Treatment for Common Urea Cycle Disorder Gains Orphan Drug Status

Urea cycle disorders are a group of inherited metabolic disorders that make it difficult for patients to remove toxic waste products.

The Food and Drug Administration (FDA) has granted Orphan Drug designation to ARCT-810 (Arcturus Therapeutics), a novel treatment for ornithine transcarbamylase deficiency (OTCD), a urea cycle disorder. 

Individuals with OTCD have mutations in the gene which leads to a non-functional or deficient ornithine transcarbamylase enzyme, and thus, the inability to convert ammonia to urea. The treatment is designed to deliver ornithine transcarbamylase (OTC) mRNA to hepatocytes, using the Company’s LUNAR lipid-mediated delivery platform

Arcturus stated that they plan to submit an Investigational New Drug application to the FDA in 2020.  

Related Articles

“Current treatment options involve life-long restriction of protein intake, nitrogen scavenger therapy as well as liver transplant for those patients that are suitable,” said Pad Chivukula, PhD, CSO of Arcturus. “Our therapeutic approach […] is designed to enable OTC-deficient patients to naturally produce healthy functional OTC enzymes in their own liver cells.”

For more information visit