DAWNZERA demonstrated significant and sustained HAE attack rate reduction and long-term disease control
Ionis Pharmaceuticals, Inc. has shared that the U.S. Food and Drug Administration has given approval for DAWNZERA (donidalorsen) to help prevent attacks of hereditary angioedema (HAE) in adults and children who are 12 years old or older. This is the first treatment that targets RNA and is approved for HAE. DAWNZERA works by targeting a protein called plasma prekallikrein (PKK), which plays a big role in the body’s inflammatory reactions that cause sudden HAE attacks. Patients can give themselves DAWNZERA 80mg using a subcutaneous autoinjector every four or eight weeks, which makes the treatment easier to manage and more flexible.
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Hereditary angioedema is a rare genetic disease that leads to repeated and severe swelling in parts of the body such as the hands, feet, face, throat, stomach, and genital areas. About 7,000 people in the U.S. are thought to live with this condition, and if not treated, it can be life-threatening. The FDA’s approval of DAWNZERA adds a new option for patients to prevent attacks, helping to reduce how often and how badly they happen, and ultimately improve their quality of life.
Brett P. Monia, Ph.D., chief executive officer of Ionis, said, “DAWNZERA is a major step forward for people living with HAE who need better treatment options. With strong and lasting effectiveness, easy way to use it, and the longest dosing schedule available, we believe DAWNZERA will become the preferred preventive treatment for many people with HAE. Importantly, the recently published switch data gives patients and doctors a clear path to switch from other preventive treatments to DAWNZERA. At Ionis, we are committed to turning exciting scientific discoveries into medicines that can really change lives. With the successful launch of our first independent medicine, TRYNGOLZA, for familial chylomicronemia syndrome, and now DAWNZERA, our second independent medicine approved in under nine months, we are proud to be delivering on that promise. We are deeply grateful to the patients, families, advocacy groups, and researchers who made this possible.”
The FDA’s approval came after positive results from the Phase 3 OASIS-HAE trial, a major, randomized, placebo-controlled study done at many global sites. The study showed that giving DAWNZERA every four weeks reduced the monthly HAE attack rate by 81% compared to a placebo during 24 weeks. When looking at the results from the second dose onwards, the attack rate dropped by 87%. The treatment also reduced moderate-to-severe attacks by around 90% over this time. Results from the ongoing OASISplus open-label study further confirmed that DAWNZERA given every eight weeks had similar benefits. After one year, DAWNZERA decreased the attack rate by 94% in both groups combined.
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The OASISplus study also looked at patients switching to DAWNZERA from other preventive treatments like lanadelumab, C1-esterase inhibitor, or berotralstat. Those who switched saw a 62% drop in attack rates over 16 weeks without more frequent attacks. In fact, 84% of these patients preferred DAWNZERA over their previous treatments, saying it offered better control of the disease, quicker administration, and fewer problems at the injection site. Across all studies, DAWNZERA had a good safety profile. The most common side effects were reactions at the site of injection, upper respiratory infections, urinary tract infections, and stomach discomfort.
Anthony J. Castaldo, CEO and chairman of the board at the U.S. Hereditary Angioedema Association and Hereditary Angioedema International, said, “As the first FDA-approved RNA-targeted therapy for HAE, DAWNZERA is a welcome advancement in treatment options for preventing attacks. Today’s approval gives people living with HAE and their doctors another important choice to match treatment with individual needs.”
Marc Riedl, M.D., M.S., clinical director of the U.S. HAEA Angioedema Center at the University of California, San Diego, and investigator in the OASIS-HAE and OASISplus trials, said, “People living with HAE have to manage this condition for their whole lives, and many still face unpredictable, painful, and dangerous attacks even with current treatments. Long-lasting effectiveness is essential for maintaining control of the disease over time. DAWNZERA is designed to meet the needs of patients by providing significant and ongoing reduction of HAE attacks, continued improvement, and less burden from treatment.”
DAWNZERA will be available in the U.S. soon. Ionis Pharmaceuticals is working to help patients through the Ionis Every Step program. This program helps patients and healthcare providers with services like help with insurance approvals, information about costs, patient education, the DAWNZERA Direct digital tool, and other resources aimed at helping patients stick with their treatment and succeed.
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