EMA Recommends Five Orphan Drugs

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The agency recommended a total of 11 drugs at its July meeting, including five orphan drugs.

The European Medicines Agency announced on July 21, 2017 that its Committee for Medicinal Products for Human Use (CHMP) recommended approval of 11 medicines at its July meeting. The approved medicines include five orphan drugs and included treatments for multiple types of cancer.

The orphan drugs the committee recommended for authorization are:

  • Verkazia (ciclosporin), which was under EMA’s accelerated assessment mechanism, for the treatment of severe vernal keratoconjunctivitis in children and adolescents.

  • Bavencio (avelumab), for the treatment of Merkel cell carcinoma

  • Lutathera (lutetium [177Lu] oxodotreotide), for the treatment of gastro-entero-pancreatic neuroendocrine tumours

  • Rydapt (midostaurin), for the treatment of acute myeloid leukaemia, aggressive systemic mastocytosis, systemic mastocytosis with associated haematological neoplasm, and mast cell leukaemia

  • Xermelo (telotristat ethyl), for the treatment of carcinoid syndrome.

CHMP also gave positive opinions to Dupixent (dupilumab), for the treatment of atopic dermatitis; Symtuza (darunavir/cobicistat/emtricitabine/tenofovir alafenamide), for the treatment of HIV infection; and Tecentriq (atezolizumab), for the treatment of locally advanced or metastatic urothelial carcinoma and of non-small cell lung cancer. Generic drugs Entecavir Accord (entecavir) and Entecavir Mylan (entecavir), both for the treatment of chronic hepatitis B; and Lacosamide Accord (lacosamide), for the treatment of epilepsy, were also given a positive opinion.

Two drugs received a negative opinion: Fanaptum (iloperidone), for the treatment of schizophrenia, and Onzeald (etirinotecan pegol), for the treatment of breast cancer with brain metastases.

Source: EMA