Biologics

FDA OKs Gene Therapy Trial for Heart Disease

FDA has approved a Tenaya Therapeutics IND for the company to begin a Phase 1b trial of TN-401, an adeno-associated virus serotype 9 (AAV9)-based investigational gene therapy product candidate for the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by mutations in the plakophilin-2 (PKP2) gene.

ARVC, which is also known as arrhythmogenic cardiomyopathy, is described as a chronic, progressive, familial disease that typically affects people before age 40. Symptoms experienced are related to ventricular arrhythmias, including palpitations, lightheadedness and fainting, and those affected are at increased risk of sudden cardiac death, Tenaya says. “PKP2 mutations are the most common genetic cause of ARVC and result in a loss of key proteins needed to maintain the structural integrity and cell-to-cell signaling of heart muscle cells,” it says.

The gene therapy is designed to address the disease’s underlying cause by delivering a fully functional PKP2 gene to “restore normal PKP2 protein levels and thereby slow disease progression and reverse the course of disease after a single dose,” the company says. “The initial dose for TN-401 in the upcoming RIDGE-1 study was associated with near maximal efficacy in our preclinical studies.” It says the clinical trial will seek to enroll up to 15 adults who have been diagnosed with PKP2-associated ARVC, have an implantable cardioverter defibrillator (ICD) and have an increased risk for arrhythmias as determined during screening by premature ventricular contraction count.

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