EU panel recommends approval of HAE treatment Dawnzera
EMA committee backs treatment for patients 12 and older
A European Medicines Agency (EMA) committee recommended that Dawnzera (donidalorsen) be approved to prevent swelling attacks in hereditary angioedema (HAE) patients 12 and older.
The positive opinion comes from the EMA’s Committee for Medicinal Products for Human Use (CHMP), which is tasked with reviewing safety and efficacy data for experimental medicines being considered for approval in the European Union. Dawnzera was recently approved in the U.S.
The European Commission, which has final say over EU drug approvals, will review the opinion. The commission isn’t required to abide by the CHMP’s opinion, but it almost always does. A final verdict from the commission is expected in early 2026, according to the therapy’s developer Ionis Pharmaceuticals.
“We believe the positive opinion from the CHMP reflects the robust clinical evidence supporting DAWNZERA and its potential to deliver a meaningful benefit to people living with HAE in the EU,” Brett P. Monia, PhD, CEO of Ionis, said in a company press release.
‘Life-changing’ potential
If approved in the EU, Dawnzera will be sold there by Otsuka Pharmaceutical, per an agreement with Ionis.
“We are encouraged by the CHMP’s positive opinion, a key milestone in advancing access to potentially life-changing treatments for the HAE community,” said Andy Hodge, president and CEO of Otsuka Pharmaceutical Europe. “We look forward to the forthcoming decision from the European Commission and remain committed to addressing the unmet needs of this patient population.”
HAE is a genetic disorder in which the body overproduces bradykinin, a signaling molecule that triggers swelling attacks. Dawnzera is an RNA-based therapy designed to reduce levels of kallikrein, the enzyme responsible for producing bradykinin. The treatment is administered by subcutaneous (under-the-skin) injection.
The CHMP’s recommendation for Dawnzera was based on data from the Phase 3 OASIS-HAE clinical trial (NCT05139810) and the extension study OASISplus (NCT05392114). The Phase 3 trial demonstrated that the therapy was more effective than a placebo in reducing the frequency of swelling attacks. Data from the extension study showed that the reduction in attack rates was sustained over the course of a year of treatment.
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