Experimental Treatment for Hereditary Angioedema Gets Fast Track Designation

According to a story from Benzinga, the pharmaceutical company BioCryst Pharmaceuticals, Inc., recently announced that its investigational product BCX7353 has been granted Fast Track Designation from the FDA for the treatment of hereditary angioedema.  BioCryst focuses on the development of treatments for rare diseases. This designation has the potential to accelerate the development of BCX7353.

About Hereditary Angioedema

Hereditary angioedema is a disorder which is characterized by episodes of severe swelling of the body. There are three main types of hereditary angioedema, and the disorder is typically caused by mutations of the SERPING1 gene. Typically, the disorder first appears in childhood. Swelling can affect the face, arms, legs, airway, and digestive tract. A swelling attack can constitute a medical emergency if the airway becomes blocked. Without intervention, swelling can appear every couple of weeks; a single episode can last for days. There are treatments available for hereditary angioedema that can help reduce the frequency of swelling and stop an episode from worsening. Generally, patients are not at risk of serious complications as long as they have access to treatment. Prevalence is about one in every 50,000 people. To learn more about hereditary angioedema, click here.

About Fast Track Designation

Fast Track Designation offers a number of advantages to the recipient company. For one, products with Fast Track Designation will receive regular collaboration and feedback from the FDA during the course of development. In addition, the product will also be eligible for priority review when the approval application is submitted, providing that the data from trials supports it.

About BCX7353

BCX7353 is meant as a once a day orally available medication that can prevent swelling attacks from hereditary angioedema and can also treat them when they occur. Currently, two clinical trials are under way to test the viability of BCX7353 in both of these situations. BioCryst estimates that they will be able to file their New Drug Application for the product in the second half of 2019.

BCX7353 could be a new opportunity for patients who would be able to take a single medication for both acute treatment and prevention of swelling attacks. Hopefully, the development of this drug will continue to progress smoothly.


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