The European Commission (EC) has granted marketing authorization for Otsuka’s Dawnzera (donidalorsen) for the routine prevention of recurrent attacks in hereditary angioedema (HAE). This marks the first RNA-targeted prophylactic therapy approved for this rare genetic condition in the European Union.
Innovative Mechanism and Clinical Efficacy Dawnzera is an antisense oligonucleotide designed to target plasma prekallikrein (PKK), which plays a pivotal role in triggering the debilitating and unpredictable swelling episodes characteristic of HAE.
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Administration: The drug is delivered via a subcutaneous autoinjector, allowing for self-administration every four or eight weeks.
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Clinical Performance: Trials demonstrated that Dawnzera reduced the monthly attack rate by 81% with four-week dosing. Long-term extension studies reported a significant 94% reduction in mean monthly attacks after one year.
Competitive Landscape and Financial Impact Dawnzera’s extended dosing schedule positions it as a convenient alternative to existing therapies, such as Takhzyro (administered every two weeks) or daily oral medications.
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Licensing Deal: Otsuka acquired the exclusive European rights to the drug from Ionis Pharmaceuticals in 2023 for $65 million upfront. The EC’s approval has triggered a $15 million milestone payment to Ionis.
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Market Scope: HAE affects approximately 15,000 individuals across Europe, many of whom require life-long prophylactic treatment to manage the potentially life-threatening condition.
Source: https://pharmaphorum.com/news/eu-approves-otsukas-long-acting-hae-drug-dawnzera