Orladeyo (berotralstat) is a plasma kallikrein inhibitor. It's taken once daily to prevent swelling attacks in the body in adults and children 12 years and older with a genetic condition called hereditary angioedema (HAE). Orladeyo (berotralstat) is the only currently available HAE medication that's taken by mouth. Side effects include stomach pain, vomiting, and diarrhea, but they should get better over time as you continue to take the medication.
Prevention of hereditary angioedema (HAE) attacks in people 12 years and older
Orladeyo (berotralstat) is a plasma kallikrein inhibitor. It blocks the activity of kallikrein, which is a protein in your blood that's involved in causing swelling and pain related to hereditary angioedema (HAE). By lowering kallikrein activity, Orladeyo (berotralstat) can help prevent pain and swelling attacks in people with HAE.
Source: DailyMed
Side effects that you should report to your care team as soon as possible:
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
Works well to prevent HAE attacks
The only HAE medication that's taken by mouth (i.e., not an injection)
Need to take only once per day
Can't treat an HAE attack that's already happening
Can cause stomach upset (e.g., stomach pain, vomiting, diarrhea)
Can't use in children under 12 years old because it hasn't been studied yet
Orladeyo (berotralstat) is only meant to be used to prevent HAE attacks, so you should have a different medication on hand to use if you experience a sudden HAE attack.
Take Orladeyo (berotralstat) exactly as prescribed and at the same time every day with food. Don't take more doses or higher doses than prescribed because this can raise your risk for side effects, including a serious and potentially life-threatening heart rhythm problem (QT prolongation).
Don't open, crush, or chew the capsules. Contact your provider if you're having trouble swallowing the capsules. You might need to switch to a different medication to help prevent HAE attacks.
Side effects like stomach upset and heartburn typically get better or go away with time as you continue to take Orladeyo (berotralstat). Talk to your provider if any side effects become too bothersome. Your provider can recommend treatment options to ease these side effects or suggest to lower your Orladeyo (berotralstat) dose.
Don't start any new medications while you're taking Orladeyo (berotralstat) without asking your provider or pharmacist first. Orladeyo (berotralstat) can interact with other medications, such as digoxin (Lanoxin) and cyclosporine (Sandimmune or Neoral). Your providers can check for interactions to make sure your medications are safe to take.
Let your provider know if you're pregnant, planning to become pregnant, or breastfeeding before you start taking Orladeyo (berotralstat). It's not known whether the medication can harm your unborn baby or pass into breast milk because it hasn't been studied.
Orladeyo (berotralstat) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Risk factors: Taking more than 150 mg of Orladeyo (berotralstat) per day | History of heart problems, slow heart rate, or other abnormal heart rhythms (arrhythmias) | Personal or family history of long QT syndrome | Abnormal electrolyte levels | Taking other medications that cause QT prolongation
Taking more than the daily recommended dose — 150 mg of Orladeyo (berotralstat) per day — can raise your risk of a serious and potentially life-threatening heart rhythm problem called QT prolongation, or long QT syndrome. This happens when your heart doesn't contract and relax when it's supposed to, which can lead to an abnormal heart rhythm.
Let your providers know about your medical history and all the medications you take so they can check your risk for QT prolongation. Your risk might be higher if you have other heart rhythm problems or if you take medications that also cause QT prolongation.
Call your provider right away if you accidentally took more than your prescribed dose of Orladeyo (berotralstat). And call 911 right away if you experience chest pain, shortness of breath, lightheadedness, or abnormal heart rhythm.
The typical dose is 150 mg by mouth once daily with food.
Your dose might differ if you have liver problems or if you're having side effects that don't go away with time.
Prevention of hereditary angioedema (HAE) attacks in people 12 years and older
Prevention of hereditary angioedema (HAE) attacks in people 6 years and older
Prevention of hereditary angioedema (HAE) attacks in people 6 years of age and older
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BioCryst Pharmaceuticals, Inc. (2023). BioCryst begins enrollment in pivotal APeX-P trial evaluating Orladeyo® (berotralstat) in pediatric patients with hereditary angioedema. Globe Newswire.
BioCryst Pharmaceuticals, Inc. (2023). Orladeyo- berotralstat hydrochloride capsule [package insert]. DailyMed.
Farzam, K., et al. (2023). QT prolonging drugs. StatPearls.
Jean-Baptiste, M, et al. (2022). The symptom experience of hereditary angioedema (HAE) patients beyond HAE attacks: Literature review and clinician interviews. Orphanet Journal of Rare Diseases.
Kiani-Alikhan, S., et al. (2023). Once-daily oral berotralstat for long-term prophylaxis of hereditary angioedema: The open-label extension of the APeX-2 randomized trial.
National Heart, Lung, and Blood Institute. (2022). Long QT syndrome.
U.S. Hereditary Angioedema Association. (n.d.). About hereditary angioedema.
Watt, M., et al. (2023). Network meta-analysis for indirect comparison of lanadelumab and berotralstat for the treatment of hereditary angioedema. Journal of Comparative Effectiveness Research.
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