Hereditary Angioedema Definition, Symptoms & Treatment: BioCryst's Genetic Disorder Drug Fails Phase 2

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BioCryst Pharmaceuticals Inc.'s drug for genetic disorder Hereditary Angioedema failed to pass the study.

Avoralstat, the drug made by BioCryst Pharmaceuticals Inc. did not reduce the number of swelling in hereditary angioedema (HAE) patients, Reuters reports.

During a trial, BioCryst administered either 500 mg or 300 mg of the avoralstat drug to patients. However, when compared to the placebo control group, the drug did not reduce the edema or recurrent swelling. The drug is meant to be taken orally as all other similar drugs are injectables.

During a press conference for the trial results, the drug company said that the failure may be because of its liquid gel capsule formulation. Additionally, it failed to retain in the proper concentration in the body as it was meant to be taken three times a day per eight hours.

"While the studies make every effort to make the patients stick with the compliance of every eight hours, it doesn't always happen," said Marc Riedl, clinical director of the Angioedema Center at the University of California, San Diego, via XConomy.

The company is currently on to the second version of their HAE drug in pill format called BCX7353. Although it is different than the first one in terms of structure, they both work similarly as they target the same enzyme called kallikrein. Their goal is to compound the new drug so it can be taken only once a day to reduce dosing errors and to prolong its effect.

HAE or Quickne disease is a rare and hereditary genetic disorder of the blood that causes recurrent swelling in the many parts of the body including the genitals, face, hands and feet to name a few. The swelling usually happens under the skin instead of on the surface, according to Medline Plus.

The condition is potentially deadly as the swelling can occur on the inside the trachea which can complicate breathing. It can also occur in the larynx as well as the intestinal tract. Treatment options for HAE are limited as antihistamines and current therapies used to treat it do not work well. Sometimes, the swelling can become worse with other treatments.

According to the US Hereditary Angioedema Association, the condition affects 1 in 10,000 or 1 in 50,000 people. Families with HAE history have a 50 percent of passing it down to their children if one parent has it. However, it is not unheard of for those who have no family history to be diagnosed with it.