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Different Endpoints Drive HAE Drugs On Road To Approval

 
• By Cathy Dombrowski

With four drugs in the space, hereditary angioedema is an unusually well-served orphan disease. But the rare condition still has no clear path for efficacy assessment.

The orphan drug condition hereditary angioedema has engendered extensive drug development activity, with multiple sponsors looking at a variety of options and a variety of endpoints to demonstrate their drugs’ efficacy. The result is four drugs in the space, each arriving with different measures of efficacy. Adding to the mix, two products were approved by the Center for Drug Evaluation and Research, while the two C1 esterase inhibitors fell under the purview of Center for Biologics Evaluation and Research as fractionated plasma products.

The hottest competition was in the quest for an indication to treat acute attacks of the disease, which can cause pain and swelling at various sites in the body, including potentially fatal laryngeal swelling

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