Experimental Treatment for Dravet Syndrome Earns Orphan Drug Designation

According to a story from Pharmaceutical Technology, the company Stoke Therapeutics experimental drug candidate, known as STK-001, has earned Orphan Drug designation from the US Food and Drug Administration (FDA). This drug, which is classified as an antisense oligonucleotide, is in development as a treatment for Dravet syndrome, a rare form of severe childhood epilepsy. Stoke’s chief medical officer, Barry Ticho, says that the company hopes that the investigational therapy will have be capable of slowing or even stopping the progression of the syndrome.

About Dravet Syndrome

Dravet syndrome is a form of severe epilepsy that usually begins by the sixth month of life. The disease is most characterized by frequent seizures that are sometimes triggered by fever or hot temperatures. In most cases, the syndrome is caused by mutations of the SCN1A gene. Most of the time, these mutations are not heritable and appear spontaneously. The predominant symptom of Dravet syndrome is seizures; varying types of seizures often occur as the disorder progresses, as well as ataxia, developmental delays, behavioral disorders that may resemble autism, and cognitive impairment. Seizures can be potentially lethal. Management strategies for Dravet syndrome may include a ketogenic diet, anticonvulsant medications, cognitive rehabilitation therapy, and cannabidiol. This rare disorder is estimated to appear anywhere from 1 in every 20,000 to 1 in every 40,000 births. To learn more about Dravet syndrome, click here.

About Orphan Drug Designation

Orphan Drug designation is typically reserved for investigational therapies that are intended to treat diseases that are considered rare. This is defined as any illness that affects less than 200,000 people in the US. To earn the designation, the drug must display advantages in efficacy and/or safety in comparison to currently available treatments. Alternatively, the drug must address a currently unmet medical need. Orphan Drug designation confers several benefits to the recipient company, such as tax breaks, the waiving of certain fees, and a period of market exclusivity lasting seven years if the drug is approved by the FDA. 

The company hopes that STK-001 will become the first approved therapy for Dravet syndrome that can modify the disease by treating the underlying cause. The mechanism of action is intended to upregulate one of the alleles of the SCN1A gene so that the NaV.1 protein can be expressed at near normal levels, reducing the chances of seizures and other symptoms.


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