Dawnzera (donidalorsen) is a medicine used to prevent attacks of hereditary angioedema (HAE). HAE is a rare genetic condition that is caused by a problem with a gene that makes a blood protein called C1 inhibitor. In most cases of HAE, you don’t have enough of this protein. In others, you have normal levels but it doesn’t work right. This causes your body to release too much of a substance called bradykinin. Bradykinin causes blood vessels to widen and swelling that can be painful and life-threatening.
During an HAE attack, swelling happens in different areas of the body, such as your face, hands, feet, gut, or throat. If it affects the gut, it can cause severe stomach pain. If throat swelling occurs, 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.
Treatment depends on the type of HAE you have and how severe it is. It focuses on avoiding and managing attacks with medicine. Because attacks can happen at any time and are hard to predict, you need to take medicine regularly to prevent attacks. You also need a rescue medicine (on-demand treatment) readily available to treat breakthrough attacks. HAE medicines work in different ways to stop or prevent your attacks by lowering or preventing too much bradykinin from being made. Dawnzera is a medicine that is approved to prevent HAE attacks in adults and children 12 and older.
How Does Dawnzera Work for Hereditary Angioedema?
Medicines called bradykinin inhibitors are used to prevent HAE attacks. The main ingredient in Dawnzera is donidalorsen, a medicine that lowers the amount of a protein called PKK (prekallikrein). PKK is made in the liver and plays a key role in swelling because it causes the release of bradykinin. By lowering PKK levels, Dawnzera used every four or eight weeks prevents too much bradykinin from being made and helps lower the number of HAE attacks.
How Should I Use Dawnzera?
Dawnzera is a liquid injected under the skin (subcutaneous). It is injected into your thighs or stomach area by you or a caregiver at home. A caregiver may also inject the medicine into the back of the upper arm. Do not inject into areas of the skin that are tender, bruised, red, or hard, or that have moles or scars. Each injection should be given at a different site on your body. Do not inject within two inches around your belly button. The injection is given once every four or eight weeks.
Dawnzera comes as a prefilled device (autoinjector) that is ready to use, so you do not need to learn how to use a syringe and needle. If your health care provider decides you can give your injections at home, they will give you instructions and teach you how to give yourself the injection.
It is important to inject the medicine properly so that you get the right dose. Your prescription should come with “Instructions for Use,” which explains how to prepare and give a dose. Ask your pharmacist or other health care provider if you are unsure how to use Dawnzera.
How Was Dawnzera Studied for Hereditary Angioedema?
A clinical study known as OASIS-HAE looked at whether Dawnzera was safe and effective for preventing HAE attacks over a six-month period. Dawnzera was compared to a placebo that contained no medicine. People in the study did not know if they were getting Dawnzera or a placebo.
The study included 90 people who were 12 or older with a diagnosis of HAE. Dawnzera or a placebo was given as an injection into the skin (subcutaneous) once every four weeks or once every eight weeks for 24 weeks. The average age of people in the study was 37. About half (53%) were female. Most were White (91%), 1% were Asian, 2% were Black or African American, 3% were American Indian or Alaska Native, and 2% were multiple or other races. To be included in the study, people were not allowed to take any other preventive medicine for HAE and had to have had at least two HAE attacks during the eight weeks before the study started. During the study, people were allowed to use their emergency medicine to treat any HAE attacks that occurred while using Dawnzera.
The main goal of the study was to look at the number of HAE attacks per month (attack rate) over six months.
What Benefits Were Seen With Dawnzera?
People who received Dawnzera during the study instead of placebo had fewer HAE attacks per month over the six-month period. Dawnzera given every four weeks reduced the number of HAE attacks by 81% as compared with a placebo. Dawnzera given every eight weeks reduced the number of HAE attacks by 55% as compared with a placebo.
During the six-month study, 53% of people who were taking Dawnzera every four weeks and 35% who were taking Dawnzera every eight weeks were completely attack-free compared to 9% who were taking a placebo.
Dawnzera reduced the amount of attacks that required on-demand treatment during the study. Dawnzera given every four weeks reduced HAE attacks requiring on-demand treatment by 92% as compared to a placebo. Dawnzera given every eight weeks reduced HAE attacks requiring on-demand treatment by 67% as compared to a placebo.
Your results may differ from what was seen in clinical studies.
How Long Does It Take to Work?
If Dawnzera is working, the number of HAE attacks you have should decrease right away. Over time, you should notice that you are having fewer attacks overall and that they are happening less often. In the study, the number of attacks began to decrease during the first month of taking the medicine. If you have less attacks while taking it every four weeks, you may be able to take it every eight 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. Dawnzera may not completely stop your attacks from occurring, but it may reduce how often they happen.
Tell your health care provider if the number of attacks you have is not getting better within the first month of using Dawnzera. You
Getting Your Medicine: What to Expect
You will receive Dawnzera from a specialty pharmacy. The drugmaker has a program called the Ionis Every Step Support Program to help support the process for you and your health care provider. You can find out more at www.dawnzera.com/resources-and-support or by calling 844-444-4305.
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 $0 for your prescription. For questions or more information, contact the drugmaker at 844-444-4305.
