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Ionis' Dawnzera Gets EU Approval for Hereditary Angioedema

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Key Takeaways

  • IONS shares rose 2.5% after the European Commission approved Dawnzera to prevent recurrent HAE attacks.
  • Ionis partnered with Otsuka ex-U.S., & is eligible for a $15M milestone payment with approval in the EU.
  • Dawnzera cut mean monthly HAE attacks by 94% at one year in the OASISplus study.

Ionis (IONS - Free Report) and its Japanese partner, Otsuka Pharmaceutical, announced that the European Commission has approved Dawnzera (donidalorsen) for the prevention of recurrent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older.

The approval was expected as the Committee for Medicinal Products for Human Use of the European Medicines Agency had issued a positive opinion in November recommending the approval of Dawnzera. The FDA had approved Dawnzera in the United States in August last year for prophylaxis to prevent attacks of HAE, a rare and potentially life-threatening genetic condition.

Ionis has a partnership with Otsuka Pharmaceutical to market Dawnzera across ex-U.S. territories. Ionis is entitled to receive $15 million as a milestone payment from Otsuka following Dawnzera’s approval in the European Union. Ionis is also entitled to earn tiered royalties of up to 30% on net product sales.

Ionis shares rose around 2.5% on Wednesday in response to the EU approval news.

Over the past year, IONS’ shares have surged 130.5% against the industry’s 3.3% fall.

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EU Nod Based on IONS’ Phase III OASIS-HAE & OASISplus Studies

Dawnzera’s EU approval was supported by robust efficacy and safety data from the phase III OASIS-HAE and OASISplus studies. Data from the studies showed that treatment with Dawnzera led to a significant reduction in the mean monthly rate of HAE attacks. The OASISplus open-label extension study showed a 94% overall reduction in mean monthly attack rate at one year.

Hereditary angioedema is a rare inherited disorder that causes repeated attacks of severe swelling in different parts of the body, including the hands, feet, face, stomach, genitals, and throat. It affects approximately one in 50,000 people globally.

Dawnzera is an RNA-targeted preventive therapy designed to reduce the production of plasma prekallikrein involved in triggering HAE attacks. It is administered by subcutaneous injections and offers long dosing intervals of four to eight weeks. Dawnzera is the first and only RNA-targeted therapy for the treatment of HAE worldwide.

Ionis’ Zacks Rank & Stocks to Consider

IONS currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are MannKind (MNKD - Free Report) , Keros Therapeutics (KROS - Free Report) , and Amicus Therapeutics (FOLD - Free Report) , each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Over the past 60 days, estimates for MannKind’s 2026 earnings per share have increased from 7 cents to 9 cents. MNKD shares have declined 4% over the past year.

MannKind’s earnings beat estimates in two quarters, missed in one and were in line in the remaining quarter, with the average surprise being 33.33%.

Over the past 60 days, 2026 loss per share estimates for Keros Therapeutics have narrowed from $3.56 to $3.36. KROS shares have surged 61.8% over the past year.

Keros Therapeutics’ earnings beat estimates in three of the trailing four quarters and missed in the remaining quarter, with the average surprise being 9098.63%.

Over the past 60 days, estimates for Amicus Therapeutics’ 2026 earnings per share have declined from 67 cents to 65 cents. FOLD shares have soared 58.3% over the past year.

Amicus Therapeutics’ earnings beat estimates in one quarter and missed in the remaining three trailing quarters, with the negative average earnings surprise being 20.21%.

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