Ionis Pharmaceuticals Trading Halted Ahead of FDA Decision on Hereditary Angioedema Drug

Ionis Pharmaceuticals' shares are halted ahead of an FDA decision on donidalorsen, a treatment for hereditary angioedema. The FDA's target action date is Thursday. Hereditary angioedema is a rare genetic condition that causes severe swelling in the body. Donidalorsen is designed to reduce the production of prekallikrein, interrupting the pathway that leads to attacks. If approved, it is expected to launch this year.

Ionis Pharmaceuticals' shares have been halted as the U.S. Food and Drug Administration (FDA) prepares to make a decision on the company's proposed treatment, donidalorsen, for hereditary angioedema. The FDA's target action date for the decision is Thursday [1].

Hereditary angioedema (HAE) is a rare genetic condition characterized by recurrent attacks of severe swelling in various parts of the body. Donidalorsen, an investigational RNA-targeted medicine, is designed to reduce the production of prekallikrein, interrupting the pathway that leads to these attacks. If approved, it is expected to launch this year [1].

The FDA's decision is crucial for Ionis Pharmaceuticals, as it has previously indicated that donidalorsen is on track to launch this year, assuming an FDA green light. The company's shares are currently halted to prevent market manipulation and ensure that investors have access to accurate and up-to-date information once the decision is made [1].

In addition to donidalorsen, there are other treatment options available for HAE patients. These include long-term prophylaxis and on-demand therapies. Long-term prophylaxis involves regular administration of medications to prevent attacks, while on-demand therapies are used to treat attacks as they occur. The treatment options available include intravenous (IV), subcutaneous, and oral medications [2].

The FDA's decision on donidalorsen could have significant implications for Ionis Pharmaceuticals and the broader biotechnology industry. If approved, it could represent a significant advancement in the treatment of HAE and potentially open up new opportunities for the company. However, if the FDA rejects the application, it could have a negative impact on the company's stock price and overall financial performance.

Investors should closely monitor the FDA's decision and the subsequent market reaction. The outcome of the FDA's review will provide important insights into the company's future prospects and the broader market for RNA-targeted medicines.

References:
[1] https://www.marketscreener.com/news/ionis-halted-as-fda-decision-looms-on-hereditary-angioedema-drug-ce7c51d3d08cf220
[2] https://www.healio.com/news/allergy-asthma/20250818/video-hae-treatment-options-expanding-becoming-more-varied