Drugs

FDA Approves Spinraza for Spinal Muscular Atrophy

FDA has approved Biogen’s Spinraza (nusinersen) for treating children and adults with spinal muscular atrophy (SMA). Spinraza, the first drug indicated for this condition, is an injection administered into the fluid surrounding the spinal cord, FDA said in an announcement.

The agency said it worked closely with Biogen and Ionis Pharmaceuticals, which developed the drug, during development to help design and implement the analysis upon which the approval was based. Spinraza’s efficacy was demonstrated in a clinical trial of 121 patients with infantile-onset SMA who were diagnosed before six months of age and who were less than seven months at the time of their first dose. The trial reportedly assessed the percentage of patients with improvement in motor milestones such as head control, sitting, ability to kick in supine position, rolling, crawling, standing, and walking. Some 40% of patients treated with Spinraza achieved improvement in motor milestones as defined in the study, while none of the control patients did.

FDA says that the most common side effects in clinical trial participants were upper respiratory infection, lower respiratory infection, and constipation. The drug’s labeling includes Warnings and Precautions for low blood platelet count and kidney toxicity.

The drug’s application was granted fast track designation, priority review, and orphan drug designation. The sponsor is being given a rare pediatric disease priority review voucher that can be redeemed in the future for priority review of a different product’s marketing application.

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