50th Orphan Medicine Receives Positive Opinion

50th orphan medicine receives positive opinion

The European Union's efforts in combating rare diseases reached a new milestone this October, with the 50th positive opinion on an 'orphan' medicine being adopted by the EMEA's Committee for Medicinal Products for Human Use (CHMP).

To date, a total of 569 medicines have been awarded orphan-designation status by the European Commission, based on recommendations of the
EMEA's Committee for Orphan Medicinal Products (COMP). Of these, 50 have now been successful in obtaining a positive EMEA opinion on marketing authorisation, with many more positive opinions expected over the coming years.

Developed specifically for the diagnosis, treatment or prevention of rare diseases, these medicines have the potential to offer relief to many thousands of European citizens suffering from chronic and often debilitating diseases who would otherwise have few-if any-treatment options available.

A listing of the 50 orphan medicines to have received a positive CHMP opinion is available here:
http://www.emea.europa.eu/pdfs/human/comp/56357508en.pdf

See also: Orphan drugs and rare diseases at a glance
http://www.emea.europa.eu/pdfs/human/comp/29007207en.pdf
EMEA website section: Orphan medicinal products
http://www.emea.europa.eu/htms/human/orphans/intro.htm