Drugs

Praxis Epilepsy Drug Study Stopped for Positive Efficacy

Praxis Precision Medicines says its experimental epilepsy drug relutrigine produced positive results in a key study, prompting an independent committee to recommend stopping the trial early for efficacy. The data come from the registrational cohort of the EMBOLD study, which is evaluating relutrigine in patients with SCN2A and SCN8A developmental and epileptic encephalopathies — ultra-rare genetic disorders marked by severe, early-onset seizures and high mortality. There are currently no approved treatments for either condition.

Praxis plans to present topline data at the American Epilepsy Society Annual Meeting in Atlanta on 12/6. It also confirms that FDA has agreed to a meeting in the coming weeks to review the study results and discuss next steps. Praxis says it will decide on the timing of an NDA submission after that discussion.

Relutrigine, also known as PRAX-562, is described as a first-in-class small molecule designed to preferentially inhibit persistent sodium currents believed to drive seizure activity in severe developmental epileptic encephalopathies. Earlier clinical and preclinical studies have shown dose-dependent seizure reduction and, in some cases, seizure freedom in patients and animal models, according to the company.

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