Orladeyo (berotralstat)
An approved treatment for Hereditary Angioedema.
Drug facts
The same compound appears under different names depending on the context. Here is how to identify Berotralstat wherever you encounter it, plus the key facts at a glance.
- Generic name
- Berotralstat
- Brand name
- Orladeyo
- Development code
- BCX7353
- Drug class
- Plasma kallikrein inhibitor (oral)
- Manufacturer
- BioCryst
- How it's taken
- 150mg by mouth once daily with food for patients 12 and older, as a capsule or, since October 2026, as 150mg oral pellets.
The first once-daily pill to prevent hereditary angioedema attacks.
- Orladeyo cut attacks by 44% versus placebo in its pivotal trial, less than the injectable antibodies, in exchange for no needles.
- Approved for prophylaxis from age 2.
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Where Berotralstat fits
The oral option for HAE prevention. Its 44% attack reduction is well below the 81-89% reported for Takhzyro, Andembry and Dawnzera, so it tends to suit patients with lower attack frequency, needle aversion, or who prefer a daily routine to a monthly injection.
How Berotralstat works
Attacks of hereditary angioedema happen when plasma kallikrein, an enzyme normally held in check by C1-inhibitor, runs free and releases bradykinin, which makes blood vessels leak. Berotralstat is a small molecule that binds plasma kallikrein and blocks it, lowering bradykinin production around the clock when taken daily. It is the same target as the antibody Takhzyro, reached with a pill instead of an injection.
Side effects and safety
- QTc prolongation at higher doses. doses above 150mg daily lengthen the QTc interval on an ECG, which is why extra doses must not be taken to treat an attack
- Stomach symptoms are the usual reason for a dose adjustment
- Dose reduced in liver impairment
The label's one warning is that doses above the recommended 150mg can prolong the QTc interval on an electrocardiogram, which is why the drug must not be taken in extra doses to treat an attack. The most common reactions, in at least 10% of patients, were abdominal pain, vomiting, diarrhea, back pain and acid reflux; stomach symptoms are the usual reason for dose adjustment. Suspected reactions can be reported to BioCryst at 1-833-633-2279.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Berotralstat
150mg by mouth once daily with food for patients 12 and older, as a capsule or, since October 2026, as 150mg oral pellets. Children aged 2 to 11 receive weight-based oral pellets from 72mg to 132mg once daily, poured into the mouth or sprinkled on soft food and taken with a meal. Dose adjustments apply for liver impairment and for persistent stomach side effects.
Starting Orladeyo? Your first 90 daysWhat happens before the first fill, the dose steps, the required tests and who to call, from the label and the drugmaker's own program.Prescribing Orladeyo? REMS, monitoring and prior authorization criteria for prescribers
Availability and cost
Only available as the brand-name product.
A daily oral small molecule for a rare disease, priced in the same range as injectable prophylaxis rather than like a conventional pill. No infusion or injection logistics, which matters for patients who cannot or will not self-inject.
Help paying for Orladeyo
With private insurance, the drugmaker's copay card can lower what you pay for Orladeyo. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE, but charity funds often can. Of the 3 charity funds for Hereditary Angioedema, 1 was open on November 9, 2026. Pick your insurance below to see which offers fit you.
- Copay help
Eligible patients may pay as little as $0 for treatment. Not valid for prescriptions paid in whole or part by federal or state programs.
For: private insurance · source - Insurance and case manager help
Dedicated patient support manager helps manage insurance, coordinates refills and shipments; clinical team including pharmacists and a nurse available 24/7.
The official page does not say who qualifies. Ask the program. · source
Good to know: Empower Patient Services is both the specialty pharmacy and the support program for Orladeyo. Copay terms are available from the Empower team. The page says patients can start regardless of insurance type but does not describe a free-drug program.
Charity funds for Hereditary Angioedema
- From a charity · The Assistance FundHereditary Angioedema (HAE) fundOpen
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
- From a charity · US Hereditary Angioedema AssociationChris Whalen HAEA Compassion Fund fundApply directly
Pays for: Travel to care for HAE-related reasons (e.g. seeing a specialist).
The foundation says: “Status not shown on page”
Clinical trial results
The APeX-2 trial (NCT03485911) randomized 120 patients aged 12 and older with type I or II HAE, each with at least 2 attacks in the first 8 weeks of run-in, to berotralstat 150mg, 110mg or placebo daily for 24 weeks. The 150mg dose reduced the attack rate by 44.2% relative to placebo (95% CI 23.0% to 59.5%), from 2.35 to 1.31 attacks per 28 days; the 110mg dose reduced it by 30%.
Reductions appeared in the first month and held through 24 weeks. Three quarters of patients had a history of laryngeal attacks and prior prophylaxis.
Development history
Developed by BioCryst as BCX7353 and approved by the FDA on December 3, 2020 as the first oral, non-steroid medicine designed specifically to prevent HAE attacks. An older male-hormone (androgen) pill, danazol, was already FDA-approved to prevent HAE attacks. The label was later extended to children aged 2 and older with a pellet formulation.
Explore Hereditary Angioedema trials
Other Hereditary Angioedema treatments
Common questions about Berotralstat
▸What is Berotralstat (Orladeyo)?
The first once-daily pill to prevent hereditary angioedema attacks. Orladeyo cut attacks by 44% versus placebo in its pivotal trial, less than the injectable antibodies, in exchange for no needles. Approved for prophylaxis from age 2.
▸How does Berotralstat work?
Attacks of hereditary angioedema happen when plasma kallikrein, an enzyme normally held in check by C1-inhibitor, runs free and releases bradykinin, which makes blood vessels leak. Berotralstat is a small molecule that binds plasma kallikrein and blocks it, lowering bradykinin production around the clock when taken daily. It is the same target as the antibody Takhzyro, reached with a pill instead of an injection.
▸What are the side effects of Berotralstat?
The label's one warning is that doses above the recommended 150mg can prolong the QTc interval on an electrocardiogram, which is why the drug must not be taken in extra doses to treat an attack. The most common reactions, in at least 10% of patients, were abdominal pain, vomiting, diarrhea, back pain and acid reflux; stomach symptoms are the usual reason for dose adjustment. Suspected reactions can be reported to BioCryst at 1-833-633-2279.
▸How is Berotralstat taken?
150mg by mouth once daily with food for patients 12 and older, as a capsule or, since October 2026, as 150mg oral pellets. Children aged 2 to 11 receive weight-based oral pellets from 72mg to 132mg once daily, poured into the mouth or sprinkled on soft food and taken with a meal. Dose adjustments apply for liver impairment and for persistent stomach side effects.
▸Is Berotralstat FDA approved?
Yes, Berotralstat (Orladeyo) is FDA approved (2020) for the treatment of Hereditary Angioedema.
▸What is Orladeyo approved for?
Orladeyo (berotralstat) is approved to prevent hereditary angioedema attacks in adults and children aged 2 and older. It was approved on December 3, 2020 as the first oral, non-steroid medicine designed specifically to prevent HAE attacks. It is not for treating an attack in progress.
▸How well does Orladeyo prevent attacks?
In the 24-week APeX-2 trial of 120 patients, 150mg daily reduced attacks by 44% compared with placebo, from 2.35 to 1.31 attacks per 4 weeks. That is a smaller reduction than the injectable antibodies report, in exchange for a once-daily pill.
▸Can I take extra Orladeyo during an attack?
No. The label states Orladeyo should not be used to treat acute attacks and that additional or higher doses are not recommended, because doses above 150mg can prolong the QTc interval on an ECG. Patients need a separate on-demand medication for attacks.
▸What are the side effects of Orladeyo?
The most common were abdominal pain, vomiting, diarrhea, back pain and acid reflux. Stomach symptoms are the usual reason for adjusting the dose. The one label warning is QTc prolongation at higher-than-recommended doses.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- BioCryst / FDA · 2026-10. ORLADEYO (berotralstat) capsules and oral pellets: US Prescribing Information. https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=ORLADEYO
- U.S. Food and Drug Administration · 2020-12-03. ORLADEYO approval, NDA 214094, December 3 2020. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=214094
- ClinicalTrials.gov. Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE (APeX-2). https://clinicaltrials.gov/study/NCT03485911