FDA approves first oral preventative therapy for kids with rare swelling disorder
(BPT) - Taking medicine to help prevent the painful and sometimes fatal swelling episodes caused by the rare genetic disorder Hereditary Angioedema, or HAE, is about to get easier for kids.
HAE causes repeat attacks of severe swelling in the body, especially the face, arms, legs, intestinal tract and airway. This can be especially dangerous for children, as their small airways can close quickly during laryngeal attacks.
Previously, children under age 12 with this rare condition only had treatment options that required injections or infusions, which can be difficult and uncomfortable for kids and their caregivers.
But the U.S. Food and Drug Administration (FDA) recently approved ORLADEYO® (berotralstat) oral pellets, the first oral preventative treatment for children ages 2 to less than 12. Taken daily, the treatment helps to prevent HAE swelling episodes, or attacks, before they start. ORLADEYO is not approved for the treatment of acute attacks.
An impactful and burdensome rare disorder
HAE often begins in childhood, and about 40% of children with HAE experience their first swelling episode by age 5. Uncontrolled HAE can significantly interfere with the ability to live a normal life, and children with HAE often miss school, extracurricular activities and other opportunities, like travel.
Regardless of age, HAE can be burdensome both physically and psychologically, and it can have a significant impact on quality of life, mental health and other developmental elements.
Treatment administration can present additional challenges
Managing the treatment of HAE can also be burdensome for families.
HAE caregiver Lisa Moore knows these challenges firsthand. Her adult son and his father were both diagnosed with HAE, and her granddaughter, Letty, age 8, was diagnosed around age 1.
"Letty was so young when she had her first attack, and it was very hard to deal with," said Moore.
There was no oral preventative treatment available at the time, and the experience of injectable medication administration was difficult for all of them. According to Lisa, Letty would run away and try to avoid it.
"The needle was the biggest thing for her," Moore said. "They had to hold her down, and it was just very hard."
A welcome oral option designed for kids
ORLADEYO oral pellets are sprinkle-like in appearance and were developed by their manufacturer, BioCryst Pharmaceuticals, to be easy for kids to take. The pellets can be poured directly into the mouth and swallowed immediately with a non-acidic liquid such as water or milk, or sprinkled over a spoonful of soft, non-acidic food.
Moore sees this as a big advance.
"To have the option of a preventative medicine in oral form for kids is wonderful. To not have to go through the trauma of being held down, being stuck with a needle, and being fearful when it comes time to have their medication will be so much easier on children and their caregivers," she said.
Clinical findings
In the largest clinical trial of a long-term preventative treatment for HAE in children ages 2 to less than 12, ORLADEYO was generally well tolerated, had a safety profile consistent with trials in people ages 12 and older, and resulted in an early and consistent decrease in HAE attacks. The most commonly reported side effect was upper respiratory infection (nasopharyngitis).
Talk with your doctor
In addition to the new oral pellet formulation, an FDA-approved capsule formulation of ORLADEYO has been available for people with HAE aged 12 and older for more than five years.
If you or someone you know has HAE, it is important to talk to a healthcare provider about treatment options. Learn more by visiting ORLADEYO.com.
Indication and Important Safety Information
WHAT IS ORLADEYO® (berotralstat)?
ORLADEYO (or-luh-DAY-oh) is a prescription medicine used to prevent attacks of hereditary angioedema (HAE) in adults and children 2 years of age and older. It is not known if ORLADEYO is safe and effective in children under 2 years of age.
It is not known if ORLADEYO is safe and effective to treat an acute HAE attack, therefore ORLADEYO should not be used to treat an acute HAE attack.
Do not take more than 1 dose of ORLADEYO per day because extra doses can cause heart rhythm problems.
IMPORTANT SAFETY INFORMATION
Before taking ORLADEYO, tell your healthcare provider about all of your medical conditions, including if you
- have liver problems or kidney problems.
- are pregnant or planning to become pregnant. It is not known if ORLADEYO can harm your unborn baby.
- are breastfeeding or plan to breastfeed. It is not known if ORLADEYO passes into your breastmilk. Talk to your healthcare provider about the best way to feed your baby while taking ORLADEYO.
Tell your healthcare provider about all of the medicines you take, including other medicines for HAE, prescription and over-the-counter medicines, vitamins, and herbal supplements.
Taking ORLADEYO with certain other medicines may affect the way other medicines work and other medicines may affect how ORLADEYO works.
Know the medicines you take and keep a list of them to show your healthcare provider and pharmacist when you get a new medicine.
What are the possible side effects of ORLADEYO?
Taking more than 1 dose of ORLADEYO per day may cause serious side effects, including heart rhythm problems. A heart rhythm problem called QTc interval prolongation can happen in people who take more than 1 dose of ORLADEYO per day. This condition can cause an abnormal heartbeat. Do not take more than 1 dose of ORLADEYO per day.
The most common side effects of ORLADEYO include abdominal pain, vomiting, diarrhea, back pain, and heartburn. These are not all of the possible side effects of ORLADEYO. For more information, ask your healthcare provider or pharmacist.
Talk to your healthcare provider for medical advice about side effects.
You are encouraged to report side effects of prescription drugs to BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or to the FDA atwww.fda.gov/medwatch or 1-800-FDA-1088.
For more information, please see the full Prescribing Information including the Patient Information.
© 2026 BioCryst Pharmaceuticals, Inc. All rights reserved. US.ORL.03216. 3/26
BioCryst and ORLADEYO are registered trademarks of BioCryst Pharmaceuticals, Inc.
Source: BrandPoint
