FDA Grants Fast Track Designation to Arcturus’ mRNA Therapeutic Candidate for OTC

Rare Daily Staff

The U.S. Food and Drug Administration has granted Fast Track designation to Arcturus Therapeutics’ ARCT-810, the company’s mRNA therapeutic candidate for ornithine transcarbamylase deficiency.

Fast Track designation is designed to facilitate development and expedite review of new therapeutics intended to treat serious or life-threatening conditions that demonstrate the potential to address important unmet medical needs. Programs granted Fast Track Designation may receive important benefits including more frequent interactions with FDA review teams and the ability to obtain rolling review of a Biologics License Application (BLA). In addition, BLA applications may receive a priority review, with the FDA aiming to complete its review within six months, instead of the standard ten months review timeline.

“This FDA designation provides several meaningful benefits that we expect will accelerate development and, hopefully, support the approval of this investigational therapy,” said Juergen Froehlich, chief medical officer of Arcturus Therapeutics.

Ornithine transcarbamylase deficiency (OTC) deficiency is a serious urea cycle disorder with a prevalence of approximately 10,000 people worldwide. A lack of OTC, a critical urea cycle enzyme in liver cells, results in high blood ammonia levels that can cause diminished cognitive ability, seizures, coma and death. The current standard of care for OTC-deficient patients is a low-protein diet, nutritional supplements and ammonia scavengers to help prevent the accumulation of ammonia. These treatments do not address the underlying cause of OTC deficiency, for which there are no FDA approved medicines.

ARCT-810 represents a novel approach to treat OTC deficiency. ARCT-810 utilizes Arcturus’ mRNA design construct and proprietary manufacturing process. ARCT-810 was developed using Arcturus’ extensive and proprietary lipid library and employs its LUNAR platform to deliver OTC mRNA to hepatocytes. ARCT-810 is an investigational mRNA medicine designed to enable OTC-deficient patients to naturally produce functional OTC enzyme in their own liver cells. Enabling patients to produce their own OTC enzyme has the potential to restore urea cycle activity, reduce the risk of metabolic decompensation, and improve quality of life.

Photo: Juergen Froehlich, chief medical officer of Arcturus Therapeutics