Amarantus Bioscience Holdings Inc (AMBS)

[Disney714]

Committee for Orphan Medicinal Products (COMP)

16 April 2015
EMA/COMP/105033/2015
Procedure Management and Business Support Division
Committee for Orphan Medicinal Products (COMP)

2.2.8
Human recombinant mesencephalic, astrocyte-derived neurotrophic factor for
treatment of retinitis pigmentosa, Clinipace GmbH - EMA/OD/327/14
[COMP co-ordinator: K. Westermark]
Following review of the application by the Committee, it was agreed to rename the active substance to
“recombinant human mesencephalic astrocyte-derived neurotrophic factor”.
The Committee agreed that the condition, retinitis pigmentosa, is a distinct medical entity and meets
the criteria for orphan designation.
The intention to treat the condition with the medicinal product containing recombinant human
mesencephalic, astrocyte-derived neurotrophic factor was considered justified based on pre-clinical in
vivo data showing protection of rods and cones as well as reduction in apoptosis of these cells.
The condition is chronically debilitating due to the development of nyctalopia and tunnel vision that
progress to total blindness.
The condition was estimated to be affecting approximately 3 in 10,000 persons in the European Union,
at the time the application was made.
The sponsor has also established that there exists no satisfactory method of treatment that has been
authorised in the European Union for patients affected by the condition.
A positive opinion for human mesencephalic astrocyte-derived neurotrophic factor, for treatment of
retinitis pigmentosa, was adopted by consensus.