Drugs

Ionis Pharma’s Alexander Disease Drug Approved

FDA has approved Ionis Pharmaceuticals’ Zanvastro (zilganersen) injection for treating Alexander disease in pediatric and adult patients — the first approved treatment for the disease and the first therapy to directly target the protein buildup that causes it.

The rare, progressive neurological disorder is caused by mutations in the gene that produces glial fibrillary acidic protein (GFAP). “When this protein is abnormal, it accumulates in the brain’s supportive cells, causing damage to the nervous system over time,” FDA says. “The disease affects less than one in a million people and can cause a wide range of serious and potentially life-threatening problems, including seizures, loss of developmental milestones, difficulty walking, muscle weakness, and increased pressure in the brain.”

Zanvastro is described as an antisense oligonucleotide, which works by reducing abnormal GFAP protein production before it can accumulate and cause further damage. It is administered as an injection into the spinal canal every three months.

FDA says that in patients aged five years and older who had measurable difficulties with walking at baseline, those treated with Zanvastro showed significantly better walking speed at 61 weeks compared to those who received no treatment.

The most common side effects reported by those on the therapy included vomiting, back pain, cough, headache, and post-lumbar puncture syndrome, the agency says.

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