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Cannabinoide als Orphan drugs

  • Kerstin Alexandra KlotzEmail author
Leitthema
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Zusammenfassung

In den letzten 5 Jahren hat der Einsatz von Cannabinoiden, allen voran Cannabidiol (CBD), in der Epilepsietherapie deutlich zugenommen. In den USA steht seit 2018 das erste FDA-genehmigte Fertigarzneimittel mit Cannabidiol zur Therapie von Dravet- und Lennox-Gastaut-Syndrom zur Verfügung, welches gleichzeitig für beide Indikationen einen Orphan-drug-Status erhielt. Durch diesen gelten erleichterte, vergünstigte und beschleunigte Zulassungsverfahren für Medikamente zur Behandlung seltener Erkrankungen. Neben randomisiert kontrollierten Studien zum Einsatz von CBD bei Dravet-Syndrom, Lennox-Gastaut-Syndrom und tuberöser Sklerose liegen inzwischen Nachweise einer antikonvulsiven Wirksamkeit von CBD auch bei anderen Epilepsieformen vor. Der aktuelle Artikel fasst die wichtigsten Informationen zum Einsatz von Cannabinoiden in der Epilepsietherapie zusammen und diskutiert die Frage, inwiefern diese mit der aktuell vorliegenden Information als Orphan drugs angesehen werden können.

Schlüsselwörter

Epilepsie Cannabidiol Pharmakotherapie Seltene Erkrankungen Tetrahydrocannabinol (THC) 

Abkürzungen

CB1

Cannabinoidrezeptor 1

CB2

Cannabinoidrezeptor 2

CBD

Cannabidiol

CBDV

Cannabidivarin

DS

Dravet-Syndrom

LGS

Lennox-Gastaut-Syndrom

THC

Delta-9-Tetrahydrocannabinol

TSC

tuberöse Sklerose

Cannabinoids as orphan drugs

Abstract

The use of cannabinoids, mainly cannabidiol (CBD) for treatment of epilepsy has clearly increased over the last 5 years. In the USA the Food and Drug Administration (FDA) approved the first purified cannabidiol preparation for treatment of Dravet syndrome and Lennox-Gastaut syndrome in 2018, which also received an orphan drug designation for these two indications. An orphan drug designation enables a simplified, beneficial and accelerated approval procedure of medications for the treatment of rare diseases. Besides randomized controlled trials on the use of CBD in Dravet syndrome, Lennox-Gastaut syndrome and tuberous sclerosis, several open label studies have meanwhile shown evidence of an anticonvulsive efficacy of CBD also for other forms of epilepsy. This article summarizes the most important preclinical and clinical information about cannabinoid treatment in epilepsy and discusses the question whether cannabinoids should be designated as orphan drugs based on the currently available information.

Keywords

Epilepsy Cannabidiol Pharmacotherapy Rare diseases Tetrahydrocannabinol (THC) 

Notes

Einhaltung ethischer Richtlinien

Interessenkonflikt

K.A. Klotz gibt an, dass kein Interessenkonflikt besteht.

Für diesen Beitrag wurden vom Autor keine Studien an Menschen oder Tieren durchgeführt. Für die aufgeführten Studien gelten die jeweils dort angegebenen ethischen Richtlinien.

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Copyright information

© Springer Medizin Verlag GmbH, ein Teil von Springer Nature 2019

Authors and Affiliations

  1. 1.Sektion Epilepsiediagnostik im Kindes- und JugendalterEpilepsiezentrum FreiburgFreiburgDeutschland

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