ΟΡΦΑΝΑ ΦΑΡΜΑΚΑ (κατά FDA) ΠΟΥ ΕΙΣΑΓΟΝΤΑΙ ΑΠΟ ΤΟ ΙΦΕΤ

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1 ΟΡΦΑΝΑ ΦΑΡΜΑΚΑ (κατά FDA) ΠΟΥ ΕΙΣΑΓΟΝΤΑΙ ΑΠΟ ΤΟ ΙΦΕΤ FDA APPROVAL [ταξινόμηση κατά δραστική ουσία (generic name)] Generic Name Trade Name Orphan Designation: Approved Orphan Indication: SPONSOR ΔΙΑΘΕΣΗ Alpha1-Proteinase Inhibitor (Human) Aminosalicylic Acid Prolastin Paser Granules For replacement therapy in the alpha-1-proteinase inhibitor congenital deficiency state. Treatment of tuberculosis infections For chronic replacement therapy of individuals having congenital deficiency of alpha1- proteinase inhibitor with clinically demonstrable panacinar emphysema. Treatment of tuberculosis Bayer Corporation Jacobus Company Baclofen Lioresal Intrathecal Treatment of intractable spasticity caused by spinal cord injury, multiple sclerosis, and other spinal diseases (including spinal ischemia, spinal tumor, transverse myelitis, cervical spondylosis, and degenerative myelopathy). Management of severe spasticity of spinal cord origin in patients who are unresponsive to oral baclofen therapy or experience intolerable CNS side effects at effective doses. Medtronic, Bendamustine Hydrochloride Treanda Treatment of chronic lymphocytic leukemia chronic lymphocytic leukemia Cephalon, ΩΣ RIBOMUSTINE Benzoate/Phenylaceta te Ammonul Treatment of acute hyperammonemia and associated encephalopathy in patients with deficiencies in enzymes of the urea cycle. Adjunctive therapy in the treatment of acute hyperammonemia and associated encephalopathy in patients with deficiencies in enzymes of the urea cycle. Medicis Corp. Betaine Cystadane Treatment of homocystinuria. Treatment of homocystinuria to decrease elevated homocysteine blood levels. Jazz s Caffeine Cafcit Treatment of apnea of prematurity. Short term treatment of apnea of prematurity in infants between 28 and less than 33 weeks gestational age. O.P.R. Development, L.P. Cysteamine Cystagon Treatment of nephropathic cystinosis. Treatment of nephropathic cystinosis in adults and children. Mylan Laboratories, Cytarabine Liposomal Depocyt Treatment of neoplastic meningitis. Intrathecal treatment of lymphomatous meningitis. Pacira s,

2 Decitabine Dacogen Treatment of myelodysplastic syndromes. for treatment of patients with myelodysplastic syndromes (MDS) including previously treated and untreated, de novo and secondary MDS of all French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia) and intermediate-1, intermediate-2, and highrisk International Prognostic Scoring System groups Eisai, Denileukin Diftitox Ontak cutaneous T-cell lymphoma persistent or recurrent cutaneous T-cell lymphoma whose malignant cells express the CD25 component of the IL-2 receptor. Eisai, Desmopressin Acetate n/a Treatment of mild hemophilia A and von Willebrand's disease. hemophilia A or von Willebrand's disease (type I) whose factor VIII coagulant activity level is greater than 5%. Aventis Behring L.L.C. ΩΣ DDAVP Digoxin Immune Fab (Ovine) Digibind Treatment of potentially life threatening digitalis intoxication in patients who are refractory to management by conventional therapy. Glaxo Wellcome Eculizumab Soliris Treatment of paroxysmal nocturnal hemoglobinuria Treatment of paroxysmal nocturnal hemoglobinuria to reduce hemolysis Alexion s, Epoprostenol Flolan Treatment of primary pulmonary hypertension. Long-term intravenous treatment of primary pulmonary hypertension in NYHA Glaxo Wellcome Class III and Class IV patients. Epoprostenol Flolan Treatment of secondary pulmonary hypertension due to intrinsic precapillary pulmonary vascular disease. For the long-term intravenous treatment of pulmonary hypertension associated with the scleroderma spectrum of disease in New York Heart Association (NYHA) Class III and Class IV patients who do not respond adequately to conventional therapy. GlaxoSmithKline LLC

3 Gemtuzumab Zogamicin Mylotarg Treatment of CD33-positive acute myeloid leukemia. CD33 positive acute myeloid leukemia in first relapse who are 60 years of age or older and who are not considered candidates for cytotoxic chemotherapy Wyeth-Ayerst Laboratories Human Fibrinogen Concentrate, Pasteurized Riastap Treatment of fibrinogen deficient patients Treatment of acute bleeding episodes in patients with congenital fibrinogen deficiency (afibrinogenemia and hypofibrinogenemia CSL Behring, LLC Hydroxocobalamin Cyanokit Treatment of acute cyanide poisoning Treatment on known or suspected cyanide poisoning Merck Sante, s.a.a. Lidocaine Patch 5% Lidoderm Patch For relief of allodynia (painful hypersensitivity), and chronic pain in postherpetic neuralgia. Teikoku Pharma USA, ΩΣ VERSATIS Mafenide Acetate Solution Sulfamylon Solution For use as an adjunctive topical antimicrobial agent to control bacterial infection when used under moist dressings over meshed autografts on excised burn wounds. Mylan Laboratories, Melphalan Alkeran For Injection multiple myeloma for whom oral therapy is inappropriate. For the palliative treatment of patients with multiple myeloma for whom oral melphalan is not appropriate. Glaxo Wellcome N- Acetylgalactosamine- 4-Sulfatase, Recombinant Human Naglazyme Treatment of mucopolysaccharidosis Type VI (Maroteaux-Lamy syndrome). For patients with mucopolysaccharidosis VI. Galsulfase has been shown to improve walking and stair-climbing capacity. BioMarin, Nitisinone Orfadin Treatment of tyrosinemia type 1. Adjunctive therapy to dietary restriction of Rare Disease tyrosine and phenylalanine in the Therapeutics, treatment of hereditary tyrosinemia type 1 Pegademase Bovine Pegaspargase Pentosan Polysulfate Sodium Adagen Oncaspar Elmiron For enzyme replacement therapy for ADA deficiency in patients with severe combined Treatment of acute lymphocytic leukemia. Treatment of interstitial cystitis. Combination chemotherapy for the treatment of patients with acute lymphoblastic leukemia who are hypersensitive to native forms of L- asparaginase. Relief of bladder pain or discomfort associated with interstitial cystitis. Enzon, Enzon, Alza Corporation Pralatrexate Folotyn Treatment of T-cell lymphoma relapsed or refractory peripheral T-cell lymphoma Allos Therapeutics,

4 Rifabutin Mycobutin Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection. Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infections. Adria Laboratories, Rifampin Rifadin I.V. For antituberculosis treatment where use of the oral form of the drug is not feasible. Hoechst Marion Roussel Sargramostim Leukine To reduce neutropenia and leukopenia and decrease the incidence of death due to infection in patients with acute myelogenous leukemia. Following induction chemotherapy in older adult patients with acute myelogenous leukemia to shorten time to Immunex neutrophil recovery and to reduce the Corporation incidence of severe and life-threatening infections and infections resulting in death. Sargramostim Leukine Treatment of neutropenia associated with bone marrow transplant, for the treatment of graft failure and delay of engraftment, and for the promotion of early engraftment. Immunex Corporation Teniposide Vumon For Injection Treatment of refractory childhood acute lymphocytic leukemia. Induction therapy in patients with refractory childhood acute lymphoblastic leukemia when used in combination with other approved anticancer agents. Bristol-Myers Squibb Research Institute Tetrabenazine Xenazine Treatment of Huntington's disease Treatment of chorea associated with Huntington's disease Prestwick s, Inc Tiopronin Thiola Prevention of cystine nephrolithiasis in patients with homozygous cystinuria. Pak, Charles Y.C. M.D. Trientine Hcl Syprine Wilson's disease who are intolerant, or inadequately responsive to penicillamine. Merck Sharp & Dohme Research ΩΣ TRIENTINE Vorinostat Zolinza Treatment of T-cell non- Hodgkin's lymphoma Treatment of cutaneous manifestations in patients with cutaneous T-cell lymphoma (CTCL) who have progressive, persistant or recurrent disease on or following two systemic therapies. Merck & Co.,

5 Zinc Acetate Galzin Treatment of Wilson's disease. For maintenance treatment of patients with Wilson's disease who have been initially treated with a chelating agent. Lemmon Company