First Subcutaneous C1 Esterase Inhibitor to Treat Rare Genetic Disease

The US Food and Drug Administration has approved Haegarda, the first C1 Esterase Inhibitor (Human) for subcutaneous (under the skin) administration to prevent Hereditary Angioedema (HAE) attacks in adolescent and adult patients.

The subcutaneous route of administration allows for easier at-home self-injection by the patient or caregiver, once proper training is received.

HAE is caused by having insufficient amounts of a plasma protein called C1-esterase inhibitor (or C1-INH), People with HAE can develop rapid swelling of the hands, feet, limbs, face, intestinal tract or airway. These attacks of swelling can occur spontaneously, or can be triggered by stress, surgery or infection.

‘The approval of Haegarda provides a new treatment option for adolescents and adults with Hereditary Angioedema,’ explained Peter Marks, MD, PhD, Director of FDA’s Centre for Biologics Evaluation and Research.

‘The subcutaneous formulation allows patients to administer the product at home to help prevent attacks.’