What Is Dawnzera, and Why Does It Matter?
Dawnzera (donidalorsen) is a new medicine approved to help prevent attacks of a rare genetic swelling disorder known as hereditary angioedema (HAE) in people aged 12 or older. Affecting about 7,000 people in the U.S., HAE causes repeated episodes of sudden, painful swelling in areas like your face, throat, stomach, genitals, or arms and legs and can become life-threatening if your airways are affected.
Dawnzera is the first medicine for HAE that works by targeting RNA, the genetic material your body uses to make certain proteins. Dawnzera blocks the production of a protein called plasma prekallikrein, which plays a key role in triggering inflammation and swelling during HAE attacks. Its targeted approach and less frequent dosing may offer a simpler way to manage HAE, especially for people who experience sudden and painful flare-ups despite treatments.
Why Was It Approved?
Dawnzera was approved because clinical studies showed that it was safe, well tolerated, and effective in reducing the number and severity of HAE attacks.
People who took Dawnzera every four weeks had about 80% fewer monthly attacks over 24 weeks compared to those taking a placebo. The reduction of attacks from baseline was 90% in those who received Dawnzera every four weeks, 83% with Dawnzera every eight weeks, and 16% in the placebo group.
In a longer-term study, people who took Dawnzera every eight weeks continued to have fewer attacks. Those who switched from other long-term HAE treatments to Dawnzera saw a 62% drop in attack frequency over 16 weeks, with no increase in flare-ups. Most people who made the switch said they preferred Dawnzera because it gave them better control of their condition, was easier to use, and caused less pain or reactions where it was injected.
The most common side effects were mild and included redness, swelling, or itching where the shot was given. Some people also had upper airway infections, urinary tract infections, or stomach discomfort. Serious allergic reactions, including anaphylaxis, were rare but have been reported.
What Do I Need to Know?
After training from your health care provider, you or your caregiver can use an autoinjector to give Dawnzera as a shot under the skin of your belly, thigh, or the back of your upper arm. The usual dose is once every four weeks, but if your condition is well controlled, your health care provider may consider switching to once every eight weeks.
Keep Dawnzera in the refrigerator, but let it sit at room temperature for 30 minutes before injecting. It can stay at room temperature for up to six weeks if needed but should be discarded if unused by then. Don’t freeze it or expose it to heat or direct sunlight. If you miss a dose, take it as soon as you remember. Then go back to your regular schedule.
Dawnzera isn’t recommended if you have moderate or severe liver problems. It hasn’t been studied in children under 12 or in people with serious kidney or liver disease. If you’ve had a serious allergic reaction to donidalorsen or any of its ingredients, you shouldn’t use this medicine. Call your health care provider right away if you notice signs of an allergic reaction, like a rash, swelling, dizziness, chest pain, or trouble breathing.
It is not known if Dawnzera harms a fetus or enters breast milk. Let your health care provider know if you are pregnant, planning to become pregnant, or breastfeeding to know your best option.