Ionis Pharma Receives FDA Approval for Dawnzera, First RNA-Targeted Therapy for Rare Genetic Swelling Disorder.

Ionis Pharmaceuticals has received FDA approval for its IONS Dawnzera (donidalorsen) to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. Dawnzera is the first RNA-targeted medicine approved for HAE and targets plasma prekallikrein, a key protein associated with acute attacks of HAE. The approval is based on positive results from the Phase 3 OASIS-HAE study, which met its primary endpoint, reducing the monthly HAE attack rate by 81% compared to placebo.

Ionis Pharmaceuticals has secured FDA approval for its Dawnzera (donidalorsen) to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 and older. This marks a significant milestone for the company, as Dawnzera is the first RNA-targeted medicine approved for HAE. The approval is based on positive results from the Phase 3 OASIS-HAE study, which demonstrated a substantial reduction in monthly HAE attack rates compared to placebo [1].

The OASIS-HAE study, a global, multicenter, randomized, double-blind, placebo-controlled trial, showed that donidalorsen, administered once every four weeks, reduced the monthly HAE attack rate by 81% compared to placebo over a 24-week period. This efficacy increased to 87% from the second dose, with a nearly 90% reduction in moderate-to-severe attacks [2]. The study also confirmed the drug's effectiveness when administered every eight weeks, with a 94% total mean attack rate reduction from baseline after one year of treatment [3].

Dawnzera's approval as an 80 mg, self-administered subcutaneous autoinjector represents a significant advancement in the treatment of HAE. The condition affects approximately 1 in 50,000 people worldwide and is characterized by recurrent swelling attacks that can be life-threatening. Dawnzera's RNA-targeting mechanism works by reducing levels of prekallikrein, a protein implicated in the onset of swelling attacks [1].

The approval of Dawnzera comes amidst a crowded field of drug research for HAE, with several therapies already available. However, Ionis believes that Dawnzera could become the preferred choice for patients due to its better efficacy, convenience, and tolerability. A majority of patients surveyed by Ionis had not yet found their best preventive option [1].

Ionis Pharmaceuticals, known for its expertise in RNA medicines, is shifting its strategy under CEO Brett Monia to launch its own drugs. Dawnzera is the second approved medicine the company will sell on its own, following Tryngolza for familial chylomicronemia syndrome [1]. Monia expressed confidence in Dawnzera's potential to become the first-line treatment for newly diagnosed patients, although the company will focus its marketing efforts on patients already taking preventive therapies [1].

The FDA approval of Dawnzera is expected to make it available in the U.S. in the coming days, with the drug's price to be disclosed during a company conference call [1]. The approval of Dawnzera represents a significant step forward in the treatment of HAE, offering patients a convenient, self-administered prophylactic option with strong and durable efficacy [2].

References:
1. [https://www.biopharmadive.com/news/ionis-donidalorsen-dawnzera-hereditary-angioedema-approval-fda/758195/](https://www.biopharmadive.com/news/ionis-donidalorsen-dawnzera-hereditary-angioedema-approval-fda/758195/)
2. [https://www.marketscreener.com/news/ionis-gets-fda-ok-of-dawnzera-in-hereditary-angioedema-ce7c50dad98bf527](https://www.marketscreener.com/news/ionis-gets-fda-ok-of-dawnzera-in-hereditary-angioedema-ce7c50dad98bf527)
3. [https://www.pharmacytimes.com/view/fda-approves-donidalorsen-as-first-rna-targeted-prophylactic-treatment-for-hereditary-angioedema](https://www.pharmacytimes.com/view/fda-approves-donidalorsen-as-first-rna-targeted-prophylactic-treatment-for-hereditary-angioedema)