Berotralstat
NA
.svg)
Drug Status
Government Approvals
US(FDA), UK(BNF)
WHO Essential Medicine
NO
Known Teratogen
No
Pharmaceutical Class
Plasma Kallikrein Inhibitor
Controlled Drug Substance
No
Berotralstat
Summary
Berotralstat is used for the long-term prevention of hereditary angioedema (HAE) attacks in adults and children aged 12 years and older. HAE is a condition that causes swelling and pain.
Berotralstat works by inhibiting an enzyme called plasma kallikrein. This enzyme is involved in the production of a substance called bradykinin, which causes blood vessels to swell. By reducing the activity of plasma kallikrein, Berotralstat helps control the overproduction of bradykinin, thereby preventing HAE attacks.
The usual daily dose of Berotralstat for adults and children aged 12 years and older is 150 mg, taken orally once daily with food. For patients with moderate or severe liver problems, the recommended dose is 110 mg once daily with food.
The most frequently reported side effects of Berotralstat include abdominal pain, vomiting, diarrhea, and headaches. These side effects are generally mild to moderate and often resolve over time.
Berotralstat should not be used for the treatment of acute HAE attacks. It should not be used by pregnant women unless clearly needed. Women of childbearing potential should use effective contraception during treatment and for at least one month after the last dose. It can interact with other medications, so inform your healthcare provider of all medications you are taking.
Indications and Purpose
How does Berotralstat work?
Berotralstat works by inhibiting plasma kallikrein, an enzyme involved in the production of bradykinin, a substance that causes blood vessels to dilate and become more permeable. In hereditary angioedema (HAE), excessive bradykinin leads to swelling and pain. By reducing plasma kallikrein activity, Berotralstat helps control the overproduction of bradykinin, thereby preventing HAE attacks.
Is Berotralstat effective?
The effectiveness of Berotralstat was demonstrated in a multicenter, randomized, double-blind, placebo-controlled study involving patients with hereditary angioedema (HAE). The study showed that Berotralstat significantly reduced the rate of HAE attacks compared to placebo. Patients taking Berotralstat experienced a 44.2% reduction in attack rate with the 150 mg dose, indicating its efficacy in preventing HAE attacks.
What is Berotralstat?
Berotralstat is used to prevent attacks of hereditary angioedema (HAE) in adults and children aged 12 years and older. It works by inhibiting plasma kallikrein, an enzyme that leads to the production of bradykinin, a substance that causes swelling and pain in HAE. By reducing plasma kallikrein activity, Berotralstat helps control the symptoms associated with HAE.
Directions for Use
For how long do I take Berotralstat?
Berotralstat is used for the long-term prevention of hereditary angioedema (HAE) attacks. The duration of use is typically ongoing, as prescribed by a healthcare provider, to maintain prophylaxis against HAE attacks.
How do I take Berotralstat?
Berotralstat should be taken orally once daily with food to help minimize gastrointestinal side effects. There are no specific food restrictions associated with Berotralstat, but it is important to follow your healthcare provider's instructions regarding diet and medication use. Always take the medication at the same time each day to maintain consistent blood levels.
How long does it take for Berotralstat to start working?
Berotralstat reaches steady-state concentrations in the body within 6 to 12 days of daily dosing. While the exact time it takes to start reducing HAE attack frequency can vary, patients may begin to notice a reduction in attack rates within the first month of treatment. It is important to continue taking the medication as prescribed to achieve the full benefit.
How should I store Berotralstat?
Berotralstat should be stored at room temperature between 68°F to 77°F (20°C to 25°C). It is important to keep the medication in its original packaging with the tamper-evident seal intact. Do not use Berotralstat if the seal is broken or missing. Always keep the medication out of reach of children.
What is the usual dose of Berotralstat?
The usual daily dose of Berotralstat for adults and children aged 12 years and older is 150 mg taken orally once daily with food. For patients with moderate or severe hepatic impairment, the recommended dose is 110 mg once daily with food.
Warnings and Precautions
Can Berotralstat be taken safely while breastfeeding?
There are no data on the presence of Berotralstat in human milk, its effects on the breastfed infant, or its effects on milk production. However, animal studies have shown that Berotralstat is present in milk. The decision to continue breastfeeding or to use Berotralstat should consider the benefits of breastfeeding and the mother's need for the medication. Consulting a healthcare provider is recommended to weigh the risks and benefits.
Can Berotralstat be taken safely while pregnant?
There are insufficient data from human studies to determine the risk of fetal harm when using Berotralstat during pregnancy. Animal studies have not shown evidence of harm to the fetus, but the drug is not recommended during pregnancy unless clearly needed. Women of childbearing potential should use effective contraception during treatment and for at least one month after the last dose. It is important to discuss potential risks with a healthcare provider.
Can I take Berotralstat with other prescription drugs?
Berotralstat is a moderate inhibitor of CYP2D6 and CYP3A4, which can affect the metabolism of other drugs. It may increase the concentration of medications metabolized by these enzymes, such as thioridazine, pimozide, cyclosporine, and fentanyl. Berotralstat is also a weak inhibitor of P-gp, which can affect drugs like digoxin. Patients should inform their healthcare provider of all medications they are taking to avoid potential interactions.
Is Berotralstat safe for the elderly?
The safety and effectiveness of Berotralstat have been evaluated in elderly patients aged 65 years and older. The results were consistent with the overall study results, indicating no specific dosage adjustments are necessary for elderly patients. However, as with any medication, elderly patients should be monitored closely for any adverse effects or interactions with other medications they may be taking.
Who should avoid taking Berotralstat?
Berotralstat should not be used for the treatment of acute HAE attacks. It is important not to exceed the recommended dose of 150 mg once daily, as higher doses can lead to QT prolongation, a heart rhythm problem. Patients with moderate or severe hepatic impairment should use a lower dose of 110 mg. Additionally, Berotralstat should be used cautiously in patients with severe renal impairment or those with risk factors for QT prolongation.
