Intellia Gets FDA Orphan Drug Status For Hereditary Angioedema Candidate

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation for Intellia Therapeutics’ (NASDAQ: NTLA) lead asset NTLA-2002, for the treatment of hereditary angioedema (HAE).

NTLA-2002 is currently being evaluated in a Phase 1/2 study in adults with Type I or Type II HAE.

NTLA-2002 is the first single-dose investigational treatment being explored in clinical trials for the potential to continuously reduce kallikrein activity and prevent attacks in people living with hereditary angioedema (HAE).

John Leonard, President and Chief Executive Officer, commented: "Orphan drug designation represents an important milestone in the development of NTLA-2002 and underscores the importance of developing innovative, new treatment options for people living with HAE. We hope to demonstrate in our ongoing clinical trial that NTLA-2002 can result in deep and sustained kallikrein activity reduction following a single dose, and potentially prevent the unpredictable swelling attacks caused by this genetic disease.”

Hereditary angioedema (HAE) is a rare, genetic disorder characterized by severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body, which can be painful, debilitating and life-threatening.

The company is planning to present interim data, including safety, kallikrein reduction and HAE attack rate details on September 16 at the 2022 Bradykinin Symposium.

FDA grants orphan designation to promote the development of a drug that is expected to have significant therapeutic advantage over existing treatments that target a condition affecting 200,000 or fewer U.S. patients annually.

Price Action : Intellia shares are trading around 1 percent higher at $60.55 on Thursday during after-hours session.