Hereditary angioedema is accompanied by persistent episodes of severe swelling of the mucous membranes and skin. The most commonly affected areas are the face, limbs, airway, and intestinal tract. These attacks may affect one or more areas at a time and subside on their own in 2 to 4 days. One-thirds of people who suffer from this condition develop a non-itchy rash, known as erythema marginatum, during an attack. HAE is of three types, I, II, and III, among which HAE type I and II are caused due to mutations in the C1NH gene.
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Current Pipeline Scenario
The HAE therapeutics pipeline consisted of 20 drug candidates in November 2017. Lanadelumab, which is being pursued by Shire PLC, was in the Phase III stage in 2017. It is a synthetically obtained peptide, which is a fully human monoclonal antibody that binds to plasma kallikrein to prevent the production of bradykinin. The drug, being developed for subcutaneous administration, is now being marketed.
Another candidate in the HAE therapeutics pipeline is BCX7353, a small molecule obtained from a synthetic source which was in the Phase II development stage in 2017 by BioCryst Pharmaceuticals Inc. Being developed to be administered orally, the candidate is a kallikrein and bradykinin inhibitor. In May 2019, the drug received the orphan drug status in Japan, and in the same month itself, Biocryst announced that it intends to submit the marketing authorization application to the European Medicines Agency in the first quarter of 2020.
Thus, it is clear that the pipeline has a bright future ahead as do people suffering from HAE.