Drugs

Roche NDA Review Extended on Spinal Disorder Drug

FDA has extended by three months its review of a Roche NDA for risdiplam and its use in treating spinal muscular atrophy (SMA). The review extension was triggered by the company’s recent submission of additional data, including data from the pivotal SUNFISH Part 2 study. The new user fee review action target date is 8/24.

In February, based on discussions with FDA, Roche submitted data which could help ensure access to risdiplam for a broad range of people living with SMA, a severe, inherited, progressive neuromuscular disease that causes devastating muscle atrophy and disease-related complications, according to the company. It is the most common genetic cause of infant mortality and one of the most common rare diseases, affecting about one in 11,000 babies, it says.

Risdiplam is a survival motor neuron-2 (SMN2) splicing modifier, Roche says. “It is designed to durably increase and sustain SMN protein levels both throughout the central nervous system and in peripheral tissues of the body,” it 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