Dawnzera wins approval in EU as preventive treatment for HAE
Injection therapy reduces risk of swelling attacks among those 12 and older
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Dawnzera (donidalorsen), an injection therapy used to reduce the risk of swelling attacks in people with hereditary angioedema (HAE), has been approved in the European Union as a preventive treatment for HAE patients 12 and older.
That decision, by the European Commission, comes shortly after the therapy was recommended for approval by a committee of the European Medicines Agency. It also follows, by just a few months, Dawnzera’s approval for the same indication in the U.S., where its now available from a specialty pharmacy.
While sold in the U.S. by its original developer Ionis Pharmaceuticals, in the EU, Dawnzera will be marketed by Otsuka Pharmaceutical under an agreement between the two companies.
“The EU approval of Dawnzera is an important milestone that reflects our ongoing commitment to bring this innovative medicine to people in need across the globe,” Brett P. Monia, PhD, CEO of Ionis, said in a press release from the two companies.
Andy Hodge, president and CEO of Otsuka, added: “We are proud of the decision from the European Commission to authorize the use of Dawnzera in HAE.”
A genetic disorder, HAE is characterized by swelling attacks caused by overactivity of the signaling molecule bradykinin. Dawnzera is an RNA-based medication designed to reduce levels of the enzyme responsible for making bradykinin, thereby decreasing levels of this signaling molecule and ultimately preventing swelling. The therapy is given by subcutaneous, or under-the-skin, injection every four or eight weeks.
Benefits of Dawnzera maintained for over 1 year in testing
Approvals of the therapy have been based primarily on findings from a Phase 3 clinical trial, OASIS-HAE (NCT05139810), involving 91 HAE patients, and its extension study, OASISplus (NCT05392114).
Results from the Phase 3 study showed that Dawnzera was better than a placebo at decreasing rates of swelling attacks, and data from the extension study showed that this reduction in rates was maintained after more than a year of treatment.
“As the first and only RNA-targeted therapy for HAE, we believe Dawnzera has the potential to become the prophylactic therapy of choice for many patients across the EU,” Monia said. “We extend our deepest gratitude to the patients, families and investigators who participated in our clinical trials and helped make this achievement possible.”
Hodge echoed those thanks, saying the approval decision “represents another key milestone in the collaboration between Otsuka and Ionis which aims to address unmet need in a challenging rare disease.” Hodge expressed appreciation for “all those at Otsuka and Ionis whose commitment has helped us introduce this medicine to patients.”
