Drugs

Ionis Plans NDA for Triglyceride Drug

Ionis Pharmaceuticals says it will file an NDA early next year after reporting Phase 3 topline results of olezarsen in people with familial chylomicronemia syndrome (FCS). The company says the trial met its primary efficacy endpoint with a statistically significant reduction in triglyceride levels with the olezarsen 80 mg monthly dose at six months compared to placebo. Additional results showed triglyceride lowering continued to improve at 12 months, and the drug showed a 100% reduction in acute pancreatitis events compared to placebo (0 events for olezarsen versus 11 events for placebo), which was a key secondary endpoint.

If approved, Ionis says olezarsen would be the first treatment in the U.S. for FCS, a rare, debilitating genetic disease that can lead to acute, potentially fatal pancreatitis attacks. FDA granted olezarsen a fast track designation for the indication earlier this year.

Olezarsen is described as a ligand conjugated antisense drug that is designed to inhibit the body’s production of apoC-III, a protein produced in the liver that regulates triglyceride metabolism in the blood.

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