Intellia Therapeutics Say EC Granted Orphan Drug Designation To NTLA-2002 For Hereditary Angioedema

(RTTNews) - Intellia Therapeutics, Inc. (NTLA) Tuesday announced that the European Commission or EC has granted orphan drug designation to its NTLA-2002 for the treatment of hereditary angioedema or HAE.

Clinical-stage genome editing company said the NTLA-2002 is an in vivo CRISPR-based investigational therapy designed to prevent potentially life-threatening swelling attacks in people with HAE. It is developed as a potential one-time treatment for people with hereditary angioedema.

Intellia CEO John Leonard said, "We are on track to complete enrollment of the Phase 2 portion of the study in the coming weeks.

Orphan drug designation in the European Union provides regulatory, financial and commercial incentives to develop therapies for rare diseases where there are either no satisfactory treatment options.

NTLA-2002 has already received orphan drug designation and RMAT designation from the FDA.

Hereditary angioedema 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.