Date: 2017-03-20
Type of information: Granting of the orphan status in the EU
Product name: Epidiolex® - cannabidiol (2-[(1R,6R)-3-Methyl-6-(1-methylethenyl)-2-cyclohexen-1-yl]-5-pentyl-1,3-benzenediol)
Compound: cannabidiol (2-[(1R,6R)-3-Methyl-6-(1-methylethenyl)-2-cyclohexen-1-yl]-5-pentyl-1,3-benzenediol)
Therapeutic area: Rare diseases
Action mechanism: cannabinol derivative/endocannabinoid modulator.
Company: GW Pharmaceuticals (UK)
Disease: Lennox-Gastaut syndrome
Latest news:
- • On February 16, 2017, the Committee for Orphan Medicinal Products (COMP) adopted a positive opinion recommending cannabidiol for designation as orphan medicinal product to the European Commission (EC) for treatment of Lennox-Gastaut syndrome.
- • On February 27, 2014, the FDA has granted orphan drug designation for Epidiolex®, GW’s product candidate that contains plant-derived cannabidiol (CBD) as its active ingredient, for use in treating children with Lennox-Gastaut syndrome (LGS), a rare and severe form of childhood-onset epilepsy. Epidiolex® is an oral liquid formulation of a highly purified extract of CBD, a non-psychoactive molecule from the cannabis plant. In November 2013, GW announced that the FDA had granted orphan drug designation for Epidiolex® in the treatment of Dravet syndrome.
- Following the successful follow-on offering completed in January 2014, GW intends to advance a full clinical development program for Epidiolex for the treatment of both LGS and Dravet syndromes. Working with leading pediatric epilepsy specialists in the United States, GW expects to commence an initial Phase 2 clinical trial for Epidiolex in the second half of 2014. This trial, if successful, is expected to lead to Phase 3 pivotal trial programs in both Dravet syndrome and LGS intended to support New Drug Applications with the FDA.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2014-02-27
Orphan status UE: 2017-03-20
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
