COMP recommends OD status for five new drugs

At a meeting in May, the Committee for Orphan Medicinal Products of the European Medicines Agency (EMEA) adopted five positive opinions recommending the following medicines for designation as orphan medicinal products to the European Commission.

These included: vincristine sulphate liposomes, from QuadraMed, as a therapy for acute lymphoblastic leukemia; beraprost sodium (modified release tablet), from Lung Rx, for the treatment of pulmonary arterial hypertension; N-(2,4-Di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carb oxamide, from Voisin Consulting, for the treatment of cystic fibrosis; and sapacitabine, from UK-based Cyclacel, for myelodysplastic syndrome and acute myeloid leukemia.

At the meeting, the Committee also welcomed Tim Cote, director of the US Food and Drug Administration's Office of Orphan Drug Development. The Committee discussed with him a future collaboration in the field of rare diseases, particularly the encouragement for cross submissions for orphan designation to the FDA and EMEA, the strengthening of communication between agencies, and the future collaboration for annual reports on development. Dr Cote also presented details on the FDA's initiative to incentivize submissions for orphan designation for tropical diseases.