Drugs

Breakthrough Status for Dravet Therapy

FDA has granted Stoke Therapeutics a breakthrough therapy designation for zorevunersen and its use in treating Dravet syndrome with a confirmed mutation in the SCN1A gene.

The designation is based on clinical data from the Phase 1/2a and open-label extension studies, which demonstrated zorevunersen’s “substantial and sustained reductions in seizure frequency and continuous improvements in multiple measures of cognition and behavior,” according to the company. Zorevunersen was generally well tolerated across the studies, it says, adding that more than 600 doses of zorevunersen have been administered to patients, with some on therapy for more than three years. “By helping the body restore naturally occurring NaV1.1 protein levels, zorevunersen is designed to treat the underlying cause of the disease,” it says.

The company says discussions with FDA and other global regulatory agencies are underway about a Phase 3 registrational study.

Zorevunersen is described as an antisense oligonucleotide that has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. It is designed to upregulate NaV1.1 protein expression by “leveraging the non-mutant (wild-type) copy of the SCN1A gene to restore physiological NaV1.1 levels, thereby reducing both occurrence of seizures and significant non-seizure comorbidities,” the company says.

Gupta Raises Concerns About Overton’s Independence, Ideology

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Overton Confirmation Remains Uncertain After Senate Hearing

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FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

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FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

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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