Drugs

Novartis to Start AVXS-101 IT Confirmatory Study

Novartis Gene Therapies says that based on an FDA recommendation it will undertake a pivotal confirmatory study to supplement the existing STRONG data on the intrathecal formulation of AVXS-101 in older patients with spinal muscular atrophy. The company says the agency acknowledged the potential of AVXS-101 IT in the patient population.

Guidance from FDA provided clarity on the path to registration for AVXS-101 IT, Novartis says, and trial design and other details are being evaluated.

The agency had placed a 10/2019 partial clinical hold on clinical trials for intrathecal administration of AVXS-101 following an AveXis (now Novartis Gene Therapies) communication to health authorities and clinical trial investigators based on findings from a small AveXis-initiated pre-clinical study in which animal findings showed dorsal root ganglia mononuclear cell inflammation, sometimes accompanied by neuronal cell body degeneration or loss.

The clinical hold did not affect the intravenous administration of Zolgensma (onasemnogene abeparvovec-xioi), also known as AVXS-101 intravenous administration.

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