Biologics

Clinical Hold on Regenxbio Hunter Syndrome Gene Therapy

FDA has placed a clinical hold on Regenxbio's investigational gene therapy RGX-121 after asymptomatic spine MRI findings were identified in five participants in a clinical trial evaluating the treatment for Hunter Syndrome or Mucopolysaccharidosis Type II (MPS II). In response, the company says it does not expect to resubmit its BLA for RGX-121 until it evaluates the findings and additional long-term data. In February, the agency issued a complete-response letter, citing fundamental concerns about the study population, choice of control, and use of a novel cerebrospinal fluid biomarker as the basis for accelerated approval.

The five participants were identified through an expanded MRI monitoring program that Regenxbio implemented several months ago following a separate clinical hold involving its RGX-111 program. The enhanced monitoring included brain and spine MRI scans.

The scans identified either a small nodule or small cystic mass in the spines of five participants who received RGX-121 approximately three to six years ago through intracisternal or intraventricular administration.

According to Regenxbio, all five participants remain clinically well and have shown overall stability or improvement on neurocognitive and neurobehavioral assessments. Investigators classified the findings as nonserious, while radiologists believe they are likely benign.

However, there is currently no clinical or pathological evidence establishing the nature or cause of the MRI findings, the company says, adding that no brain nodules or masses were detected in any of the brain MRI scans.

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