This site is intended for health professionals only
Tuesday 25 June 2019
Share |

Orphan drug committee proposes new designations

During its first meeting in 2012, the Committee for Orphan Medicinal Products (COMP) adopted a total of 16 recommendations for orphan designation, including for a number of substances intended for the treatment of diseases for which no orphan designation has ever been granted, and a substance intended for the treatment of a neglected tropical disease.

The COMP recommended the granting of orphan designation for carbetocin for the treatment of Prader-Willi syndrome and for sialic acid in the treatment of hereditary inclusion body myopathy. These were the first orphan designation opinions for the treatment of these rare conditions.

The COMP recommendations will now be sent to the European Commission for the adoption of a decision, which will be published on the agency’s website. 

Once an orphan designation has been granted, sponsors have access to certain incentives during the development of the medicine.

Committee for Orphan Medicinal Products

Ads by Google

You are leaving

You are currently leaving the Nursing in Practice site. Are you sure you want to proceed?


Respect for nurses: Sign up to our e-petition TODAY

The Nursing in Practice Respect campaign is now live! Over the coming months, we're set to highlight the vital contribution and efforts of primary care and community care nurses throughout the UK.

As part of our campaign, Nursing in Practice is looking to call on parliament to set up a debate to celebrate the vital work that you do.



Calling all primary care nurses! 'Like' our Nursing in Practice Facebook page to enter our free draw to win a £25 M&S voucher