Drugs

Priority Review for Ionis Alexander Disease Drug

FDA accepts for priority review an Ionis Pharmaceuticals NDA for zilganersen, an experimental treatment for Alexander disease. The agency set a user fee review action target date of 9/22.

Alexander disease is described as a progressive neurological disorder that primarily affects children and leads to severe motor and cognitive decline, loss of independence, and premature death. There are currently no disease-modifying therapies available.

Ionis says the submission is supported by data from a pivotal clinical trial in which zilganersen demonstrated statistically significant and clinically meaningful stabilization in patients’ gait speed, as measured by the 10-Meter Walk Test at 61 weeks. The treatment also showed favorable results across secondary measures, including communication, adaptive function, and symptoms such as seizures and sleep disturbances, with a generally well-tolerated safety profile.

Zilganersen is an antisense oligonucleotide therapy designed to reduce production of glial fibrillary acidic protein, a substance that accumulates abnormally in patients with the disease and drives neurological damage, according to the company.

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

Read more

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