According to a story from Angioedema News, an investigational product from BioCryst Pharmaceuticals called BCX7353 was able to quickly subdue swelling attacks in hereditary angioedema in clinical trials. In addition, the drug appears to reduce the frequency in which symptoms appear. The medication can also reduce the need for using rescue medications. These capabilities were recorded in results from the company’s Phase 2 clinical trials.
About Hereditary Angioedema
Hereditary angioedema is a genetic disorder which is characterized by chronic episodes of swelling that can affect multiple areas of the body. The condition is caused by mutations affecting the HAE gene. Swelling attacks generally occur every two weeks or so; they can usually last for several days. Swelling may affect the limbs, digestive tract, face, and airway, with blockage of the airway being the most dangerous complication. Vomiting and abdominal pain may accompany attack as well if the digestive tract is involved. Treatment involves reducing the likelihood for attacks to appear and preventing them from worsening when they do. Hereditary angioedema is typically only life-threatening if left untreated. Prevalence of the condition is estimated to be around one in 10,000 to one in 50,000, at least in the US and Canada. To learn more about hereditary angioedema, click here.
About BCX7353 And The Phase 2 Trial
BCX7353 appears to be a treatment that can have dual purpose role for hereditary angioedema patients, since it can stop attacks quickly and prevent them from occurring. The experimental treatment is being formulated as a liquid for stopping attacks in progress and as a capsule for preventing the appearance of attacks in the long term. In the trial, BCX7353 was able to bring down swelling in about an hour after administration with a 750mg dosage. The effect of the drug was able to last for 24 hours. Treatment with this drug also allowed patients to halt their normal standard of care at a rate of 31.6 percent. Side effects were minor and included headache, diarrhea, and nasal inflammation.
Results from dosing in both the 250mg and 500mg are expected to be released in early 2019. Overall, these results are quite encouraging, and if the therapy gains approval, it will be the first medication for hereditary angioedema that can be used for treating attacks and preventing attacks over the long term.