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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.

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