Dravet syndrome is a rare form of epilepsy that typically manifests in infancy or early childhood. According to the National Institutes of Health (NIH), initial symptoms often present as either focal or generalized convulsive seizures that begin before the age of 15 months, with many cases occurring before the age of one. These initial seizures are frequently prolonged and involve one side of the body, followed by a switch to the other side. Additional seizure types may emerge after the age of 12 months, displaying a wide range of characteristics.

Children with Dravet syndrome typically exhibit normal development during the first few years of life. However, as seizures become more frequent, their developmental progress may slow, causing them to fall behind their peers. The majority of Dravet syndrome cases are linked to a mutation in the SCN1A gene, which is crucial for the proper functioning of brain cells. While seizures associated with Dravet syndrome are challenging to manage, they can be mitigated to some extent through the use of anticonvulsant medications.

In this article, we will discuss the latest guidelines on Dravet syndrome as outlined by the Dravet Syndrome Foundation. Currently, there are no US-specific guidelines, only international consensus. Additionally, we will highlight the three most recent medications that have been approved for the treatment of Dravet syndrome. This article will focus on the guideline and the FDA updates that have recently emerged.

Part 1 – Part 1 – Current Dravet Syndrome Guidelines

As previously mentioned, there are currently no established guidelines in the United States regarding Dravet syndrome. Therefore, we have included a current guideline/international consensus as a point of reference:

  • The Diagnosis and Treatment of Dravet Syndrome
    • Dravet Syndrome Foundation
    • Publication: May 1, 2022
    • The Dravet Syndrome Foundation funded an International Consensus Panel Study on the diagnosis and management of Dravet syndrome. The guidelines include clinical presentation, EEG and MRI findings, genetic testing, first and second-line treatments for seizures, and other aspects of treatment.
Part 2 – Recent FDA Approvals

Below, are the recent medications and therapies recently approved by the FDA that we anticipate being incorporated into future US guidelines:

  • FINTEPLA® (fenfluramine)
    • UCB, Inc.
    • FDA approved for Dravet syndrome on June 25, 2020
    • FINTEPLA is an amphetamine derivative indicated for the treatment of seizures associated with Dravet syndrome and Lennox-Gastaut syndrome in patients 2 years of age and older.

  • DIACOMIT (stiripentol)
    • Biocodex, Inc.
    • FDA approved for Dravet syndrome on August 20, 2018
    • DIACOMIT is an anticonvulsant for use as an adjunctive treatment with clobazam in patients who are 6 months of age and older and weighing 7 kg or more. There is no clinical data to support the use of DIACOMIT as monotherapy in Dravet syndrome.

  • EPIDIOLEX® (cannabidiol)
    • Jazz Pharmaceuticals, Inc.
    • FDA approved for Dravet syndrome on June 25, 2018
    • EPIDIOLEX is a prescription pharmaceutical formulation of highly-purified, marijuana plant-derived cannabidiol (CBD) indicated for the treatment of seizures associated with Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex.

Thank you for taking the time to review the current Dravet syndrome guidelines, as well as the recent major changes and FDA approvals that have occurred. We value your feedback and would like to hear your suggestions for future topics to be covered in our guideline series. Please feel free to contact us with any ideas or questions you may have. We look forward to hearing from you.

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