Stoke Therapeutics’ STK-001 Receives FDA’s Orphan Drug Designation for Dravet Syndrome

Shots:

• The US FDA granted Orphan Drug Designation to Stoke’s STK-001 with its expected onset of P-I/II clinical study and IND submission to the US FDA in H1’19 and H1’20 respectively
• The FDA’s ODD is granted supporting the development of drugs for safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. Additionally, the designation also provides various development incentives, including tax credits for qualified clinical testing, an exemption from FDA application fees, and a 7yrs. period of marketing exclusivity in the US, if approved
• STK-001 is an investigational antisense oligonucleotide candidate, designed to upregulate NaV1.1 protein expression from the non-mutant (wild type) copy of the SCN1A gene restoring physiological NaV1.1 level in patients with Dravet syndrome

Click here to­ read full press release/ article | Ref: Stoke Therapeutics| Image: LinkedIn