Drugs

Praxis Relutrigine NDA Accepted for Rare Pediatric Epilepsies

FDA has accepted for priority review a Praxis Precision Medicines NDA for relutrigine, an investigational therapy targeting rare genetic epilepsies. The agency set a user fee review action target date of 9/27.

Relutrigine is being developed to treat developmental and epileptic encephalopathies (DEEs) caused by mutations in the SCN2A and SCN8A genes — severe neurological disorders that begin in infancy or early childhood and are associated with frequent, difficult-to-control seizures and high mortality, according to the company. There are currently no approved targeted therapies for these conditions.

The NDA is supported by data from the Phase 2 EMBOLD study, which was halted early after an interim analysis indicated efficacy, following a recommendation from an independent data monitoring committee.

According to Praxis, relutrigine demonstrated improvements in motor seizure control in a heavily pretreated patient population, with some patients achieving sustained seizure freedom. The therapy has also been generally well-tolerated across clinical studies to date.

Relutrigine is designed as a first-in-class small molecule that selectively inhibits persistent sodium current, a mechanism believed to drive neuronal hyperexcitability and seizures in severe epileptic disorders, Praxis says. Preclinical studies have shown dose-dependent seizure control, including complete suppression in animal models of SCN2A- and SCN8A-related disease.

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