Stoke Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its lead product candidate, STK-001. STK-001 is an investigational new treatment that has the potential to be the first disease-modifying therapy to address the underlying genetic cause of Dravet syndrome. It is designed to upregulate NaV1.1 protein expression from the non-mutant copy of the SCN1A gene to restore physiological NaV1.1 levels, thereby reducing seizures and significant non-seizure comorbidities.
The FDA Office of Orphan Products Development mission is to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diesease. Orphan drug designation entitles recipients to various development incentives since there is no reasonable expectation that the cost of developing and making available a drug will be recovered from sales for such drugs.
Read the full press release here.