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FDA grants ARC-AAT orphan drug status to treat liver disease
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ARC-AAT has been granted orphan drug designation by the FDA for the treatment of liver disease associated with alpha-1 antitrypsin deficiency, according to Arrowhead Research Corporation, manufacturer of the drug.
ARC-AAT works to knock down the alpha-1 antitrypsin gene transcript and reduce the hepatic production of the mutant AAT protein, according to Arrowhead. Alpha-1 antitrypsin deficiency (AATD) is a rare genetic disorder associated with liver disease in children and adults and pulmonary disease in adults.
The orphan drug designation will provide incentives for sponsors to develop products for rare diseases. These include marketing exclusively, qualifying for various development incentives and 7 years of orphan exclusivity when a new drug application is approved.
“Receiving orphan drug designation is an important milestone in the development of ARC-AAT, which we think is a very promising program aimed at providing a better option for patients with liver disease associated with alpha-1 antitrypsin deficiency,” Bruce D. Given, MD, chief operating officer of Arrowhead, said in the release. “The Orphan Drug Act provides important incentives for sponsors to develop drugs that treat rare diseases and we look forward to more engagement with the FDA as the development of ARC-AAT progresses.”
ARC-AAT is currently being investigated in part B of a phase 1 clinical trial in patients with PiZZ genotype AATD, according to the release. The trial is a double-blind, single dose-escalation first-in-human study to evaluate the safety, tolerability and pharmacokinetics of ARC-AAT and the effect on circulating AAT levels. The study has been enrolling in dose cohorts of six participants each, with participants randomly assigned a single intravenous injection of either ARC-AAT or placebo. The study consists of two parts; part A is in healthy volunteers, which has been completed. The study evaluates participants for 28 days following dosing, with additional follow-up if needed every 2 weeks until AAT levels return to baseline.
The mean estimated prevalence of AATD in the U.S. is approximately 100,000 patients and is also a cause of pediatric liver disease with an estimated prevalence in children of approximately 20,000 patients, according to the release.
Disclosures: Given is employed by Arrowhead.