EC awards Aplidin orphan drug status

Orphan drug status has been granted by the European Commission to Aplidin for the treatment of acute lymphoblastic leukaemia (ALL), following adoption of a positive opinion by the Committee for Orphan Medicinal Products of the European Agency for the Evaluation of Medicinal Products (EMEA).

This designation would grant 10-year marketing exclusivity in Europe to Spanish biopharmaceutical company for this indication subject to successful regulatory approval.

Aplidin is PharmaMar's second marine-derived compound and is currently in Phase II clinical trials for solid tumours including renal, colorectal, head and neck, pancreatic and non-small cell lung cancers, as well as medullary thyroid carcinoma. Early studies have shown Aplidin to be very selective for acute lymphoblastic leukaemia cells over normal blood cells at concentrations that have already been easily achieved in the blood plasma of patients participating in clinical trials for other indications. In addition, current clinical data suggest that Aplidin has a favourable side-effect profile, making it an ideal drug candidate either for patients with poor bone marrow reserves or in combination with other drugs, especially as it is not haematotoxic.

'With this orphan drug designation the European Commission recognises the urgent need for new therapies for ALL,' said Jose Maria Fernandez Sousa-Faro, chairman of PharmaMar. 'Aplidin has shown very promising preclinical results in this indication and the orphan drug status will support our efforts to develop Aplidin as an effective treatment for this difficult tumour that is the most frequent in childhood.'