Arcturus Therapeutics (NASDAQ:ARCT) received FDA orphan drug designation for ARCT-810 for the treatment of ornithine transcarbamylase (OTC) deficiency.
OTC deficiency is an inherited metabolic disorder that results in the accumulation of ammonia, a neurotoxin and liver toxin, in patients’ blood.
ARCT-810 is a messenger RNA (mRNA) replacement therapy designed to help a patient’s own liver cells produce OTC.
“OTC deficiency is a life-threatening genetic disease. Current treatment options involve life-long restriction of protein intake, nitrogen scavenger therapy as well as liver transplant for those patients that are suitable,” Pad Chivukula, Arcturus’ CSO, said in a statement.
“By intervening directly in the underlying disease process, ARCT-810 has the potential to be a transformative mRNA therapy for these patients,” he added.
Arcturus plans to submit an IND application for ARCT-810 to the FDA in the first quarter of 2020.
