Takhzyro (lanadelumab-flyo) is a biologic medicine used to prevent hereditary angioedema (HAE) attacks in people who are 2 years or older. During an HAE attack, swelling under the skin happens in different areas of the body. While swelling in the throat area is not particularly common, when throat swelling does occur, it can be deadly because it can close off your airway. Certain situations or triggers can cause these attacks to happen. Common triggers for attacks include stress, surgery, medical or dental procedures, infections, and tiredness. People with HAE have to take medicine to prevent these attacks from occurring and need to have a rescue medicine readily available to treat any breakthrough attacks.
In 2018, Takhzyro was approved by the FDA as the first monoclonal antibody that is injected under the skin to help prevent swelling attacks in people with HAE. Monoclonal antibodies are medicines made in a lab that can directly target pathways in your body that lead to your symptoms.
How Does Takhzyro Work for HAE?
Most people who have HAE have a problem with a protein called C1 inhibitor — they either do not make enough of the protein or they make enough of the protein, but it does not work right. When levels of the C1 inhibitor protein are too low, it causes the body to make too much of another protein called plasma kallikrein. Plasma kallikrein is responsible for releasing yet another protein in the body called bradykinin.
When there is too much bradykinin in the body, it causes your blood vessels to leak fluid into the surrounding tissues. This causes the symptoms of HAE, including swelling and pain, often in the face, hands, feet, genitals, stomach, or throat. Takhzyro is an antibody that attaches to kallikrein in your blood. Once attached to kallikrein, Takhzyro blocks it from working, thus lowering the amount of bradykinin that your body makes. This helps prevent the fluid leakage that causes swelling, lowering the number and severity of HAE attacks that you have.
How Do I Use Takhzyro?
Takhzyro is given as an injection under your skin (subcutaneous) once every two or four weeks. Each injection can be given by a health care provider. After proper training, you or a caregiver may also give the injection. Anyone less than 12 years old should have their injection given by a caregiver or health care provider. The dose that you will get depends on how old you are.
Your prescription for Takhzyro will either come as a vial or prefilled syringe containing a single dose. Before you use it, the medicine should be stored in the refrigerator between 36 F and 46 F (2 C and 8 C) and kept in the carton to protect it from light. Take Takhzyro out of the refrigerator 15 minutes before you are ready to give your dose so it can warm up to room temperature.
If your prescription comes in a vial, you will need to draw the dose up into a syringe according to the medicine’s Instructions for Use. Once the dose is ready in the syringe, it may be kept at room temperature for up to two hours or in a refrigerator for up to eight hours. If the prepared syringe is not used after two hours at room temperature or eight hours under refrigeration, it must be thrown away.
If you are giving the dose to yourself, Takhzyro can be injected under the skin in your stomach area (abdomen) or thigh. Doses given by a caregiver can be injected into these locations or into the upper arm. Avoid injecting Takhzyro within two inches of your belly button; do not inject into areas that are bruised, swollen, scarred, or painful. Inject Takhzyro into a different place each time you get a new dose. This is called rotating the injection site.
After you have used your dose of Takhzyro, make sure to throw away any used needles and syringes in an FDA-approved sharps container. You can find out more about sharps disposal in the state you live in on the FDA website at fda.gov/safesharpsdisposal.
How Was Takhzyro Studied for HAE?
The safety and effectiveness of Takhzyro for the prevention of HAE were studied in 125 people who were 12 years or older in a clinical study called the HELP study. Another small study called the SPRING study looked at its safety and effectiveness in younger children who were less than 12 years old. To be enrolled in either of these studies, people had to have HAE caused by a problem with the C1 inhibitor protein (classified as HAE type 1 or 2) and had to have a certain number of HAE attacks over time. In the HELP study, people with at least one HAE attack per month were enrolled. In the SPRING study, children had to have at least one HAE attack every three months.
In the HELP study, people were either given Takhzyro or a placebo that contained no medicine, both injected under the skin. People who got Takhzyro were given one of three different doses: 150 mg or 300 mg every four weeks or 300 mg every two weeks. In the SPRING study, everyone got Takhzyro 150 mg injections every two or four weeks.
In the HELP study, investigators looked at how many HAE attacks people had over a 26-week time frame to see how well Takhzyro worked. The main goal of the SPRING study was to make sure Takhzyro was safe to use in younger children.
What Were the Characteristics of People in the HELP and SPRING Studies?
In both studies, nearly all of the people enrolled (more than 90%) had type 1 HAE, which happens when you do not make enough C1 inhibitor. This is the most common type of HAE. Other characteristics are described for each study below.
HELP study (≥ 12 years old)
- The average age was about 41 years.
- Most people were female (70.4%).
- Most people were White (90%), 8% were Black, and 2% were Asian.
- About two thirds of people had a history of HAE attacks that caused swelling in the airways.
- About half the people had received previous long-term treatment to prevent HAE attacks.
- At the start of the study, people had an average of about four attacks per month.
SPRING study (2 to <12 years old)
- The average age was about 8 years.
- More than half the people (57.1%) were female.
- Most people were White (95.2%), and 4.8% were Asian.
- About one quarter of the people in the study had a history of HAE attacks that caused swelling in the airways.
- A total of 14% of the people had received previous long-term treatment to prevent HAE attacks.
- At the start of the study, people had about two HAE attacks per month.
What Benefits Were Seen With Takhzyro?
In the HELP study, Takhzyro lowered the number of HAE attacks overall, the number of HAE attacks that required rescue medicine, and the number of moderate-to-severe HAE attacks compared to placebo (see Table below).
Percent reduction with Takhzyro compared to placebo | |||
|---|---|---|---|
Takhzyro 150 mg injected every 4 weeks | Takhzyro 300 mg injected every 4 weeks | Takhzyro 300 mg injected every 2 weeks | |
| Number of HAE attacks | 76% | 73% | 87% |
| Number of HAE attacks requiring rescue medicine | 81% | 74% | 87% |
| Number of moderate or severe HAE attacks | 70% | 73% | 83% |
Although the main focus of the SPRING study was safety, investigators also reported reductions in the number of HAE attacks with Takhzyro.
Your results may differ from what was seen in clinical studies.
What Is Known About the Long-Term Effectiveness of Takhzyro?
People who completed the 26-week HELP study and other people with HAE who did not participate in the HELP study could enroll in a long-term study of Takhzyro called the HELP OLE study. Everyone in the HELP OLE study was treated with Takhzyro 300 mg injected under the skin every two weeks for an average of about two and a half years. Throughout the extended study period, regular use of Takhzyro lowered the rate of HAE attacks as compared to baseline (the time period before people started using Takhzyro).
Your results may differ from what was seen in clinical studies.
How Long Does It Take to Work?
It takes Takhzyro about 10 weeks to build up in your body. Over time, you should notice that you are having fewer attacks overall and that when they happen, they are less severe. If you have less attacks while taking it every two weeks, you may eventually be able to take it every four weeks.
Do not stop using this or any other medicines for HAE without talking to your health care provider first, even if your symptoms have gotten better. Your symptoms may get worse again if you stop or change any of your medicines. Takhzyro may not completely stop your attacks from occurring, but it may reduce how often they happen.
How Can I Prevent and Manage Side Effects?
In studies, pain, redness, or bruising in the areas where Takhzyro was injected were the most common side effects. Other common side effects were airway infections (such as the common cold), headache, rash, dizziness, diarrhea, and muscle pain.
If you have injection site pain, redness, or irritation after injecting Takhzyro, ask your health care provider if an over-the-counter (OTC) pain medicine or antihistamine may help. They can tell you if you can safely take an OTC medicine and when to take it to help reduce any pain, itching, or irritation you may have at the injection site.
To lower your risk of upper airway infections, wash your hands often, scrubbing with soap and water for at least 20 seconds. Clean and disinfect surfaces that may be touched by someone who’s sick, such as countertops, handrails, and doorknobs. Avoid close contact with other people who are sick.
If you get dizzy after your injection, try sitting or lying down until you feel better. Make sure to sit or stand up slowly when you are dizzy to avoid falls. Staying well-hydrated by drinking plenty of fluids can help with dizziness and other symptoms such as headache and muscle pain. Staying hydrated is also important if you have diarrhea during treatment.
There may be other side effects of Takhzyro that are not listed here. Contact your health care provider if you think you are having a side effect of a medicine. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088 (800-332-1088). In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.
Getting Your Medicine: What to Expect
You will receive Takhzyro from a specialty pharmacy. The drugmaker has a Patient Support Program to help support the process for you and your health care provider. You can find out more at www.takhzyro.com/getting-takhzyro/patient-support or by calling 866-888-0660.
Insurance Approval. Your insurance may require approval for using this medicine, also called a prior authorization. The insurance company reviews the prescription from your health care provider to make sure it is covered.
Cost Support. There is a cost support program from the drugmaker that may allow you to pay as low as $0 for your prescription. For questions or more information, contact the drugmaker at 866-888-0660.
