Ionis Pharmaceuticals announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion on its RNA‑targeted therapy DAWNZERA (donidalorsen) for the routine prevention of hereditary angioedema (HAE). The opinion, issued on November 14, 2025, recommends the drug for approval in the European Union and has been forwarded to the European Commission, where a final decision is expected in the first quarter of 2026.
The CHMP opinion covers patients aged 12 years and older and is based on robust Phase 3 data from the OASIS‑HAE and OASISplus studies. These trials demonstrated a significant reduction in mean monthly HAE attack rates and a favorable safety profile, with common adverse reactions limited to injection‑site reactions, upper respiratory tract infections, urinary tract infections, and abdominal discomfort.
DAWNZERA received U.S. FDA approval in August 2025, and the EU endorsement would extend its reach to an estimated 1 in 50,000 people across Europe, a prevalence that aligns with global estimates for HAE. Ionis and partner Otsuka Pharmaceutical hold exclusive rights to market the drug in Europe and the Asia Pacific region, positioning the company to launch the product quickly once regulatory approval is finalized.
The regulatory milestone comes at a time when Ionis’s financial performance is strengthening. In Q3 2025, the company reported revenue of $157 million, up 17% year‑over‑year, and a loss per share of $0.80 versus an expected $1.22. The earnings beat was driven by strong sales of its wholly‑owned medicines, particularly TRYNGOLZA, and by royalty income from partnered products.
In response to the positive CHMP opinion and the Q3 earnings beat, Ionis raised its 2025 revenue guidance to $875 million–$900 million, up from the prior $825 million–$850 million range. CEO Brett P. Monia said the opinion “reflects the robust clinical evidence supporting DAWNZERA and its potential to deliver a meaningful benefit to people living with HAE in the EU.” CFO Elizabeth L. Hougen noted that the company’s revenue growth “demonstrates the strength of our portfolio and the confidence of our partners.”
Ionis’s antisense platform continues to underpin its strategy of delivering first‑in‑class therapies. The company aims to achieve cash‑flow breakeven by 2028 and expects substantial positive cash flow thereafter, driven by the commercial launch of DAWNZERA and continued growth of its existing product portfolio. The CHMP opinion therefore represents a key step in Ionis’s roadmap to expand its rare‑disease footprint in Europe and to accelerate the commercial potential of its antisense‑based medicines.
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