Committee for Orphan Medicinal Products (COMP): Monthly report from the October 2011 meeting
Reference Number: EMA/COMP/811210/2011Summary:
The Committee for Orphan Medicinal Products held its 127th plenary meeting on 5-7 October 2011.
This month the COMP delivered for a first time ever a positive opinion on orphan designation for the treatment of Leigh syndrome, for which no authorised treatments exist in the EU. This syndrome is a very rare and severe disease caused by mutations in mitochondrial respiratory enzymes, leading mainly to neurological deficits and a poor survival for these patients.