Drugs

Hunter Syndrome Gene Therapy Accepted for Review

FDA has accepted for priority review a Regenxbio BLA seeking accelerated approval for gene therapy RGX-121 (clemidsogene lanparvovec) for treating mucopolysaccharidosis II (MPS II), also known as Hunter syndrome. The agency has set a user fee review action target date of 11/9.

“Supported by positive biomarker data and long-term outcomes, RGX-121 has the potential to be a first-in-class gene therapy that could dramatically transform the MPS II treatment landscape and reduce the significant burden patients and families currently face with weekly enzyme replacement therapy,” the company says.

It describes RGX-121 as a potential one-time adeno-associated virus therapeutic that is designed to deliver the iduronate-2-sulfatase (IDS) gene to the central nervous system (CNS). “Delivery of the IDS gene within cells in the CNS could provide a permanent source of secreted iduronate-2-sulfatase (I2S) protein beyond the blood-brain barrier, allowing for long-term cross-correction of cells throughout the CNS,” it says.

MPS II is a recessive disease caused by a deficiency in the lysosomal enzyme I2S, leading to an accumulation of glycosaminoglycans, including heparan sulfate, in tissues that eventually results in cell, tissue, and organ dysfunction, Regenxbio 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