Orphan & Rare Disease Medicines

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  Hematology & Rare Blood Disorders

  Deferasirox – Transfusion-dependent Iron Overload Disorders

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  Hematology & Rare Blood Disorders

  Eculizumab – Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical HUS

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  Neurology & Neuromuscular Disorders

  Edaravone – ALS

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  Infectious & Tropical Rare Diseases

  Eflornithine – African Trypanosomiasis

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  Metabolic & Genetic Disorders

  Elosulfase alfa – Mucopolysaccharidosis Type IV (Morquio Syndrome)

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  Hematology & Rare Blood Disorders

  Eltrombopag – Immune Thrombocytopenia (ITP)

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  Infectious & Tropical Rare Diseases

  Foscarnet – Cytomegalovirus (CMV) Infection in Immunocompromised Patients

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  Metabolic & Genetic Disorders

  Idursulfase – Mucopolysaccharidosis Type II (Hunter Syndrome)

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  Oncology (Rare Cancers)

  Imatinib – Chronic Myeloid Leukemia (CML)

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  Metabolic & Genetic Disorders

  Imiglucerase / Velaglucerase alfa – Gaucher Disease

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  Pulmonology & Cardiology

  Ivacaftor – Cystic Fibrosis

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  Metabolic & Genetic Disorders

  Laronidase – Mucopolysaccharidosis Type I (Hurler Syndrome)