People with hereditary angioedema (HAE), a rare genetic disorder that can cause sudden and potentially life-threatening swelling, can now treat these episodes with a pill instead of taking injected or infused medicines.
The U.S. Food and Drug Administration (FDA) has approved Ekterly (sebetralstat), the first on-demand oral treatment for acute HAE attacks in adults and children 12 and older, its developer KalVista Pharmaceuticals said in a statement.
HAE attacks that cause swelling in the abdomen, larynx (voice box), face, and extremities can develop with little warning and may quickly become life-threatening if people don’t take rescue medicines to reverse the symptoms and halt the progression of the episode, says Timothy Craig, DO, a professor of medicine at Penn State University in University Park, Pennsylvania, who has helped write international guidelines for HAE management.
Because sebetralstat is a pill taken by mouth, it’s “easier to carry, easier to have in your pocket, and easier to take,” Dr. Craig says.
While the new drug works about as well as older medicines for HAE attacks, such as the generic injected drug icatibant, Ekterly may have better results for some patients if its convenience translates into faster treatment, Craig says.
This matters, because people with HAE are prone to spontaneous attacks even when they regularly take medications designed to prevent them, says Jonathan Bernstein, MD, a professor at the University of Cincinnati College of Medicine who worked on research studies of garadacimab for hereditary angioedema.
“An oral treatment might permit HAE patients to take the medication earlier after the onset of symptoms, which would be more impactful at preventing the progression of the attack,” Dr. Bernstein says.
What Is Hereditary Angioedema (HAE)?
People with HAE usually need two types of medicines: preventive drugs that help reduce the risk of attacks, and on-demand or rescue drugs that treat attacks when they do happen.
How Fast Does Sebetralstat Work for HAE Attacks?
In a late-stage clinical trial, researchers randomly assigned participants 12 years and older with HAE to take either a 300 milligram (mg) or 600 mg dose of sebetralstat or a placebo for acute attacks. During the trial, 110 participants experienced a total of 264 acute HAE attacks that they treated with one of the Ekterly doses or with a placebo pill.
With the new drug, HAE symptoms started to improve after about 1.6 to 1.8 hours, compared with around 6.7 hours with the placebo, according to study findings published in the New England Journal of Medicine (NEJM).
Up to about half of people on the drug experienced complete symptom relief within 24 hours, compared with about 27 percent on the placebo.
Headaches were the most common side effects for Ekterly, and no serious adverse events were reported during the study.
In the real world, some people might need to take a second dose of rescue medicines like Ekterly to achieve sufficient symptom relief, Craig says. Some might also need to go to a hospital if swelling occurs in the airways and restricts breathing.
“If you have an airway attack, you should treat yourself as soon as you can and go directly to an emergency room or call 911,” Craig says.
How Much Will Ekterly Cost?
Ekterly was immediately available when it received FDA approval, according to its developer, KalVista. While the company didn’t disclose pricing information, the drug should be free for many patients with private health insurance who can get discounts from the drug company, Craig says.
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