Method for the treatment of dravet syndrome

Patent metadata

Field	Value
Publication number	US-2016136114-A1
Application number	US-201615003161-A
Country	US
Kind code	A1
Filing date	Jan 21, 2016
Priority date	May 3, 2013
Publication date	May 19, 2016
Grant date	—

Abstract

A method of treating and/or preventing Dravet Syndrome in a patient such as a patient previously diagnosed with Dravet Syndrome, by administering an effective dose of fenfluramine or its pharmaceutically acceptable salt to that patient. Dravet Syndrome patients are typically children under the age of 18 and are treated at a preferred dose of less than about 0.5 to about 0.01 mg/kg/day.

First claim

1 .- 20 . (canceled)

21 . A method of treatment, comprising:
administering to a patient diagnosed with Dravet Syndrome a therapeutically effective dose of a 5-HT2C agonist; and allowing the 5-HT2C agonist to stimulate 5-HT2C receptors in the patient and thereby reduce seizures in the patient.

22 . The method of claim 21 , wherein the 5-HT2C agonist is fenfluramine.

23 . The method of claim 21 , wherein the therapeutically effective dose is less than about 0.5 mg/kg/day to about 0.01 mg/kg/day.

24 . The method of claim 23 , wherein said patient is aged 18 or less.

25 . The method of claim 21 , wherein the therapeutically effective dose is administered in a dosage form selected from the groups consisting of oral, injectable, transdermal, inhaled, nasal, rectal, vaginal and parenteral.

26 . The method of claim 22 , wherein the therapeutically effective dose is fenfluramine or a pharmaceutically acceptable salt thereof is co-administered with one or more co-therapeutic agents.

27 . The method of claim 21 , wherein the patient exhibits a mutation in a gene selected from the group consisting of SCN1A, SCN1B, SCN2A, SCN3A, SCN9A, GABRG2, GABRD and PCDH19.

28 . The method of claim 26 , wherein the one or more co-therapeutic agents is an anticonvulsant.

29 . The method of claim 26 , wherein the one or more co-therapeutic agents is selected from the group consisting of carbamazepine, ethosuximide, fosphenytoin, lamotrigine, levetiracetam, phenobarnitol, progabide, topiramate, stiripentol, valproic acid, valproate, verapamil, and benzodiazepines such as clobazam, clonazepam, diazepam, ethyl loflazepate, lorazepam, and midazolam or a pharmaceutically acceptable salt thereof.

Assignees

• Univ Leuven Kath
• Univ Hospital Antwerp

Inventors

• Ceulemens Berten
• Lagae Lieven

Classifications

• A61P43/00

  Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00 · CPC title

• A61P25/08

  Antiepileptics; Anticonvulsants · CPC title

• A61K31/5513 Primary

  1,4-Benzodiazepines, e.g. diazepam {or clozapine} · CPC title

• A61K31/135

  having aromatic rings {, e.g. ketamine, nortriptyline (methadone A61K31/137)} · CPC title

• C12Q2600/156

  Polymorphic or mutational markers · CPC title