Preventive Treatment for Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE) is a rare genetic condition. It causes sudden attacks of severe swelling in the body. These attacks can happen in the stomach, face, or throat. Swelling in the throat can block breathing and become life-threatening.¹

People with HAE lack a vital protein. This protein is called C1 esterase inhibitor. Without it, a chemical called bradykinin builds up. Bradykinin causes fluid leakage, which leads to swelling.¹

Goals of preventive treatment

The main goal of HAE care is to help people live normal lives. Doctors want to reduce the number of swelling attacks. They also want to make the attacks less severe. Another goal is to prevent dangerous throat swelling before it starts, as any swelling to the throat and airway can be life-threatening.²

Treatment plans often include long-term prophylaxis (preventive treatment). This approach means taking medicine regularly to stop attacks. Regular care can give people peace of mind and improve daily living.²

How preventive treatments work

Preventive medicines work by fixing the chemical imbalance in the body. Some drugs replace the missing C1 esterase inhibitor protein. Other drugs block the proteins that create bradykinin. Blocking these proteins stops the chain reaction that causes tissue swelling.^2-4

These medicines are taken in different ways. Some are injections given under the skin. Others are given through an intravenous line into a vein. There are also oral pills that people can swallow daily.^2-4

Examples of preventive drugs for HAE

The US Food and Drug Administration (FDA) has approved several preventive drugs. These options help people manage their condition effectively. Each drug targets a specific part of the swelling pathway. Your doctor can help you choose the best option.^3,4

Examples include:^3,4

• Andembry® (garadacimab-gxii) – the first drug that targets activated factor XII. This protein sits at the top of the swelling cascade. The medicine is a once-monthly subcutaneous injection. It is approved for adults and children aged 12 and above.
• Cinryze® (C1-esterase inhibitor - human) – a plasma-derived protein replacement. It is a medicine given intravenously. It helps maintain proper C1-esterase inhibitor protein levels to prevent attacks. It is approved for adults and children aged 6 and older.
• Dawnzera™ (donidalorsen) – an RNA-targeted medicine. It targets the genetic messenger to stop the production of prekallikrein in the liver. People can self-administer this drug every 4 or 8 weeks. It uses a single-use autoinjector. It is approved for adults and children aged 12 and older.
• Haegarda® (C1 esterase inhibitor - human) – another C1 esterase inhibitor replacement. This medicine is given as a subcutaneous injection under the skin. People usually inject this treatment 2 times each week. It provides steady protection against swelling episodes. It is approved for adults and children aged 6 and older.
• Orladeyo® (berotralstat) – an oral preventive treatment option for HAE. This drug blocks plasma kallikrein to stop swelling before it begins. It is approved as a capsule for adults and children aged 12 and older. For children aged 2 to 11, small oral pellets are recommended and should be taken with food.
• Takhzyro® (lanadelumab) – a monoclonal antibody that targets plasma kallikrein. This enzyme plays a role in releasing bradykinin. The medicine is a subcutaneous injection given every 2 or 4 weeks. It is approved for people aged 2 years and older.

Possible side effects of preventive treatments

Side effects can vary depending on the specific treatment you take. Common side effects include pain or redness at the injection site. Some people experience headaches, colds, or stomach upset. These symptoms are usually mild and resolve quickly.^3,4

These are not all the possible side effects of preventive treatments. Talk to your doctor about what to expect when taking preventive medicines. You should also call your doctor if you have any changes. Contact your healthcare provider for any issues that concern you.

Other things to know before starting treatment

Before beginning treatment for HAE, tell your doctor about your health conditions. Share a list of any other drugs, vitamins, or supplements you take. This list should include over-the-counter drugs. Some medicines can interact with your HAE treatment.