Biologics

Cell Therapy for Pediatric Spinal Muscular Atrophy OK’d

FDA has approved an AveXis BLA for Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), a severe form of SMA and a leading genetic cause of infant mortality.

FDA says that the rare genetic disease is caused by a mutation in the survival motor neuron gene. The gene encodes the survival motor neuron (SMN) protein, which is found throughout the body and critical for the maintenance and function of motor neurons. “Motor neurons in the brain and spinal cord control muscle movement throughout the body,” an agency release says. “If there is not enough functional SMN protein, then the motor neurons die, leading to debilitating and often fatal muscle weakness.”

Zolgensma is an adeno-associated virus vector-based therapy that targets the cause of SMA. “The vector delivers a fully functional copy of human SMN gene into the target motor neuron cells,” FDA says. “A one-time intravenous administration of Zolgensma results in expression of the SMN protein in a child's motor neurons, which improves muscle movement and function, and survival of a child with SMA. Dosing is determined based on the weight of the patient.”

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