CHMP approves four orphan disease drugs

The EMA’s Committee for Medicinal Products for Human Use (CHMP) has now approved four orphan disease drugs – Sanofi’s Cablivi (caplacizumab), Ultragenyx’s Mepsevii (vestronidase alfa), Baxalta’s Veyvondi (vonicog alfa), and Jazz Pharmaceuticals’ Vyxeos (liposomal daunorubicin/cytarabine).

Cablivi, for acquired thrombotic thrombocytopenic purpura (aTTP), is the first therapeutic to be recommended for approval in Europe for aTTP, and thoughts are that it could make a real difference and achieve sales of around $500m each year. Current treatment is the expensive process of plasma exchange with immunosuppressive drugs that have serious side effects. Mepsevii, a mucopolysaccharidosis type VII drug for the incredibly rare lysosomal storage disorder, works as an enzyme replacement therapy. Veyvondi, for the treatment of von Willebrand disease, should be used as when standard therapy desmopressin is ineffective or contra-indicated. Vyxeos is for the treatment of newly-diagnosed acute myeloid leukaemia (AML), and will become the first chemotherapy option in the EU specifically for therapy-related AML or AML with myelodysplasia-related changes if the European Commission approves it.