Drugs

Ionis NDA for Hereditary Angioedema Attacks

FDA has accepted for review an Ionis Pharmaceuticals NDA for donidalorsen, an RNA-targeted treatment for preventing hereditary angioedema (HAE) attacks in patients aged 12 years and older. The agency has set a user fee review action target date of 8/21/2025.

The submission is based on data from several clinical trials, including the Phase 3 OASIS-HAE and OASISplus studies, as well as an ongoing Phase 2 extension study. The studies demonstrated that donidalorsen, administered either monthly or bi-monthly, significantly reduced HAE attack frequency, according to the company.

Data presented recently at the 2024 American College of Allergy, Asthma & Immunology Annual Scientific Meeting showed that donidalorsen reduced HAE attack rates by an average of 96% from baseline, Ionis says. Across the studies, donidalorsen was well-tolerated with most adverse events mild or moderate, primarily consisting of injection site reactions, it adds.

Hereditary angioedema, a rare genetic condition, causes recurring episodes of severe swelling in different parts of the body, such as the hands, feet, genitals, abdomen, face, and throat. This swelling can be painful and life-threatening in severe cases, according to the company. Donidalorsen is said to work by targeting prekallikrein, a protein involved in triggering the swelling pathway.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome