New Treatment for Rare Genetic Condition Leave a comment


(RxWiki News) The US Food and Drug Administration (FDA) has approved a new treatment for a rare genetic disease.

This new treatment is called Haegarda (C1 esterase inhibitor (human)). The FDA approved it to prevent swelling and painful attacks in those with hereditary angioedema (HAE).

This medication is only meant to prevent attacks, so it will not treat an acute HAE attack that is already occurring, the FDA noted.

HAE occurs when a protein called C1 does not work the way it should or there is not enough of it in the blood. This protein helps control inflammation and parts of the immune system.

Those with HAE can develop swelling of the hands, feet, limbs, face, and even the intestinal tract or airway. The condition is passed on in families and affects between 6,000 and 10,000 Americans, according to the FDA. 

Haegarda is available as an injection to be administered under the skin twice weekly (every three or four days).

“The approval of Haegarda provides a new treatment option for adolescents and adults with hereditary angioedema,” said Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, in a press release. “The subcutaneous formulation allows patients to administer the product at home to help prevent attacks.”

Common side effects include injection-site reactions, allergic reactions, and swelling of the nasal passages and throat.

The FDA granted approval of Haegarda to CSL Behring.



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