Drugs

FDA Lifts Clinical Hold on Pompe Gene Therapy

FDA has lifted a clinical hold against Astellas Pharma and its FORTIS Ph1/2 clinical trial evaluating AT845, an adeno-associated virus gene replacement therapy in adults with late-onset Pompe disease. The hold was placed 6/27/2022 following a report of peripheral sensory neuropathy in one of the trial participants.

At the time, FDA told Astellas that it did not have sufficient information to assess the risks to subjects and requested additional information about the adverse event, according to the company. Astellas says it is now completing the clinical and regulatory activities necessary to resume dosing in the clinical trial.

AT845 is designed to use an AAV8 vector under a muscle-specific promotor to deliver a functional copy of the GAA gene to treat Pompe Disease patients. “AT845 is being investigated to determine whether it can deliver a functional GAA gene that is efficiently transduced to express GAA directly in tissues affected by the disease, including skeletal and cardiac muscle,” Astellas says. The primary endpoints in FORTIS include change in muscle GAA protein expression and enzyme activity from baseline, it says, while secondary endpoints are evaluating improvements in respiratory, endurance and quality of life measures.

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