Intellia Therapeutics receives European Union orphan drug designation for NTLA-2002, an investigational in-vivo CRISPR genome editing treatment for Hereditary Angioedema

Intellia Therapeutics today announced that the European Commission (EC) has granted orphan drug designation to NTLA-2002 for the treatment of hereditary angioedema (HAE).

NTLA-2002 is an in vivo CRISPR-based investigational therapy designed to prevent potentially life-threatening swelling attacks in people with HAE.

John Leonard, MD, Intellia President and Chief Executive Officer, said: “The European Union orphan drug designation for NTLA-2002 represents another important milestone for Intellia as we continue to make rapid progress in the development of a novel, potential one-time treatment for people with hereditary angioedema. We are on track to complete enrollment of the Phase 2 portion of the study in the coming weeks, which will bring us one step closer to our goal of delivering a potentially life-changing treatment for people who suffer from this serious and debilitating disease.”

Orphan drug designation in the European Union (EU) is granted by the EC based on a positive opinion issued by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products. To qualify for orphan drug designation, a candidate therapy must be intended for the treatment, prevention or diagnosis of a life-threatening or chronically debilitating disease that occurs in not more than five in 10,000 people in the EU. The designation provides regulatory, financial and commercial incentives to develop therapies for rare diseases where there are either no satisfactory treatment options or significant benefit to those affected by the disease.

(Source: Intellia)