METHOD FOR THE TREATMENT OF DRAVET SYNDROME

• US 20160136114A1
• Filed: 01/21/2016
• Published: 05/19/2016
• Est. Priority Date: 05/03/2013
• Status: Active Grant

• Current Assignee: Katholieke Universiteit Leuven, University Hospital Antwerp
• Original Assignee: Katholieke Universiteit Leuven, University Hospital Antwerp
• Inventors: Lagae, Lieven, Ceulemens, Berten
• Granted Patent: US 9,603,815 B2
• Time in Patent Office: Days
• US Class Current: 1/1
• CPC Class Codes: A61K 2300/00 Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
• CPC Class Codes: A61K 31/135 having aromatic rings , e.g. ketamine, nortriptyline methadone A61K31/137
• CPC Class Codes: A61K 31/137 Arylalkylamines, e.g. amphetamine, epinephrine, salbutamol, ephedrine or methadone
• CPC Class Codes: A61K 31/19 Carboxylic acids, e.g. valproic acid salicylic acid A61K31/60
• CPC Class Codes: A61K 31/36 Compounds containing methylenedioxyphenyl groups, e.g. sesamin
• CPC Class Codes: A61K 31/551 having two nitrogen atoms, e.g. dilazep
• CPC Class Codes: A61K 31/5513 1,4-Benzodiazepines, e.g. diazepam or clozapine
• CPC Class Codes: A61K 45/06 Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
• CPC Class Codes: A61P 25/08 Antiepileptics; Anticonvulsants
• CPC Class Codes: A61P 43/00 Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
• CPC Class Codes: C12Q 1/6883 for diseases caused by alterations of genetic material
• CPC Class Codes: C12Q 2600/156 Polymorphic or mutational markers

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.

Claims

1-20. -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.