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Orphan Drug Designations and Approvals List As of 12-02-2013 Governs January 1, 2014

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Orphan Drug Designations and Approvals List as of 12‐02‐2013 Governs January 1, 2014 ‐ March 31, 2014 Row Designation Generic Name Trade Name Orphan Designation Contact Company/Sponsor Num Date Treatment of Follicular Lymphoma, Small Lymphocytic Lymphoma, Lymphoplasmacytic Lymphoma, Splenic Marginal Zone Lymphoma, Extranodal Marginal Zone B‐cell Lymphoma of Mucosa‐ Associated Lymphoma Tissue (MALT), and Nodal Marginal Zone Lymphoma (Collectively Indolent B‐cell Non‐Hodgkin's 1 bendamustine hydrochloride Treanda 11/26/2013 Lymphoma) Cephalon, Inc. Treatment of subjects at risk of developing myelosuppression after a radiological or nuclear 2 Filgrastim Neupogen 11/20/2013 incident Amgen, Inc. adeno‐associated viral vector containing the human NADH Treatment of Leber Hereditary 3 Dehydrogenase 4 Gene n/a 11/20/2013 Optic Neuropathy Gen Sight Biologics Treatment of liver transplant recipients with reestablished fibrosis to delay the progression to cirrhosis and 4 emricasan n/a 11/20/2013 end stage liver disease Conatus Pharmaceuticals Inc. Page 1 of 262 Orphan Drug Designations and Approvals List as of 12‐02‐2013 Governs January 1, 2014 ‐ March 31, 2014 5‐((4‐bromo‐2‐ fluorophenyl)amino)‐4‐fluoro‐N‐(2‐ hydroxyethoxy)‐1‐methyl‐1H‐ Treatment Stage IIB‐IV Novartis Pharmaceuticals 5 benzo[d]imidazole‐6‐carboxamide n/a 11/19/2013 melanoma. Corporation Treatment of Hepatitis Delta 6 Lonafarnib n/a 11/19/2013 Virus (HDV)infection Eiger Biopharmaceuticals, Inc. Treatment in Stage IIB‐IV melanoma positive for the Novartis Pharmaceuticalues 7 MEK162 + LGX818 n/a 11/19/2013 BRAF mutation. Corporation Methyl [(2S)‐1‐{[4‐(3‐{5‐chloro‐2‐ fluoro‐3‐ [(methylsulfonyl)amino]phenyl}‐1‐ isopropyl‐1H‐pyrazol‐4‐yl)‐2‐ Treatment of Stage IIB‐IV pyrimidinyl]amino}‐2‐ melanoma postive for BRAF Novartis Pharmaceuticals 8 propanyl]carbamate n/a 11/19/2013 mutation Corporation autologous Prevention of graft rejection CD4+CD25hiFoxP3+regulatory T following solid organ 9 cells n/a 11/19/2013 transplantation iREG Medical AB 10 human haptoglobin n/a 11/19/2013 Treatment of sickle cell disease BioProducts Laboratory Limited recombinant human alpha‐ glucosidase conjugated with synthetic bis‐mannose‐6‐ 11 phosphate‐Man6 glycan n/a 11/19/2013 Treatment of Pompe Disease Genzyme, a Sanofi Company 2‐[(1R,6R)‐3‐Methyl‐6‐(1‐ methylethenyl)‐2‐cyclohexen‐1‐yl]‐ 12 5‐pentyl‐1,3‐benzenediol n/a 11/14/2013 Treatment of Dravet syndrome. GW Pharma Ltd. 13 eltrombopag Promacta 11/8/2013 Treatment of aplastic anemia GlaxoSmithKline LLC Treatment of Acute Limb 14 Poloxamer 188 n/a 11/8/2013 Ischemia Mast Therapeutics, Inc Page 2 of 262 Orphan Drug Designations and Approvals List as of 12‐02‐2013 Governs January 1, 2014 ‐ March 31, 2014 live attenuated bioengineered Treatment of human papilloma Listeria monocytogenes virus‐associated head and neck 15 immunotherapy n/a 11/4/2013 cancer Advaxis, Inc. Prevention of Hepatitis C monoclonal antibody directed at recurrence in patients receiving MassBiologics‐University of MA 16 hepatitis C virus E2 glycoprotein n/a 11/4/2013 liver transplantation Medical School 3‐chloro‐4‐fluorophenyl‐[4‐fluoro‐ 4‐[[(5‐methylpyrimidin‐2‐ ylmethyl)amino]methyl]piperidin‐ 17 1yl]methanone n/a 10/25/2013 Treatment of Rett syndrome Neurolixis, Inc. PEGylated recombinant anti‐ Treatment of cystic fibrosis Pseudomonas aeruginosa PcrV patients with Pseudomonas 18 Fab' antibody n/a 10/25/2013 aeruginosa lung infections KaloBios Pharmaceuticals, Inc. Treament of acute radiation 19 TXA127 n/a 10/25/2013 syndrome US Biotest, Inc. Treatment of onchocerciasis Janssen Research & Development, 20 flubendazole n/a 10/25/2013 caused by Onchocerca volvulus LLC 21 isavuconazonium sulfate n/a 10/25/2013 Treatment of zygomycosis Astellas 22 oleylphosphocholine n/a 10/25/2013 Treatment of leishmaniasis Dafra Pharma International nv poly(lactide‐co‐glycolide) Treatment of acute Cour Pharmaceutical Development 23 carboxylated microparticle n/a 10/25/2013 encephalitis syndrome Company, Inc. recombinant human monoclonal IgM antibody targeting glucose Treatment of multiple 24 regulated protein 78 n/a 10/25/2013 myeloma Patrys Ltd. Page 3 of 262 Orphan Drug Designations and Approvals List as of 12‐02‐2013 Governs January 1, 2014 ‐ March 31, 2014 For the treatment of gastric Kadcyla cancer including Ã?Æ?Ã?Â??Ã?¢âÂ??â¬Ã? gastroesophageal junction 25 trastuzumab emtansine ¡Ã?Æ?âÂ?¬Å¡Ã?Â??ã?® 10/25/2013 cancer. Genethech, Inc. Treatment of occulopharyngeal 26 trehalose Cabaletta 10/25/2013 muscular dystrophy BIOBLAST PHARMA LTD. Gallium‐68 (DOTA0‐Phel‐ The management of Society of Nuclear Medicine & 27 Tyr3)octreotide n/a 10/23/2013 neuroendocrine tumors Molecular Imaging glycyl‐L‐2‐methylprolyl‐L‐glutamic Treatment of Fragile X 28 Acid n/a 10/23/2013 Syndrome Neuren Pharmaceuticals, Ltd. Treatment of diffuse large B‐ 29 ibrutinib n/a 10/23/2013 cell lymphoma Pharmacyclics, Inc. Treatment of hypoxic‐ischemic encephalopathy to prevent 30 phenobarbitol sodium injection n/a 10/23/2013 siezures in neonates Fera Pharmaceuticals, LLC Treatment of patients with 31 sodium fusidate n/a 10/23/2013 prosthetic joint infections Cempra Pharmaceuticals, Inc. Treatment of acute myeloid 32 tigecycline n/a 10/23/2013 leukemia. Stem Cell Therapeutics Corporation fusion protein analog with recombinant human growth hormone (rhGH) at once‐a‐month Treatment of growth hormone 33 dosing n/a 10/16/2013 deficiency Versartis, Inc. 34 human IgG1k monoclonal antibody n/a 10/16/2013 Treatment of systemic sclerosis MedImmune Treatment of hepatocellular 35 mTOR kinase inhibitor (CC‐223) n/a 10/16/2013 carcinoma Celgene Corporation 36 olaparib n/a 10/16/2013 Treatment of ovarian cancer AstraZeneca Pharmaceuticals LP Treatment of hepatocelular Daiichi Sankyo Pharma 37 tivantinib n/a 10/16/2013 carcinoma Development Page 4 of 262 Orphan Drug Designations and Approvals List as of 12‐02‐2013 Governs January 1, 2014 ‐ March 31, 2014 6‐amino‐5‐chloro‐N‐[1R)‐1‐[5‐[[[5‐ hloro‐4‐(trifluoromethyl)‐ 2pyridinyl]amino]carbonyl]‐2‐ thiazoyl]ethyl]‐4‐ Treatment of stage IIb‐IV 38 pyrimdinecarboxamide n/a 10/15/2013 melanoma Millennium Pharmaceuticals, Inc. human monoclonal antibody againt human interleukin 13 (IL‐ Treatment of eosinophilic Novartis Pharmaceuticals 39 13) n/a 10/15/2013 esophagitis Corporaton Treatment of Waldenstrom's 40 ibrutinib n/a 10/15/2013 macroglobulinemia Pharmacyclics, Inc. Treatment of chronic lymphocytic leukemia and 41 idelalisib n/a 10/15/2013 small lymphocytic lymphoma Gilead Sciences, Inc Treatment of splenic marginal 42 idelalisib n/a 10/15/2013 zone lymphoma Gilead Sciences, Inc. Treatment of nodal marginal 43 idelalisib n/a 10/15/2013 zone lymphoma. Gilead Sciences, Inc. Treatment of extranodal 44 idelalisib n/a 10/15/2013 marginal zone lymphoma Gilead Sciences, Inc. Treatment of non‐small cell lung cancer that is anaplastic 45 LDK378 n/a 9/27/2013 lymphoma kinase(ALK)‐postive Novartis Pharmaceuticals Corp. self‐complimentary adeno‐ associated virus vector, serotype 9, packaging the full lenght GAN Treatment of Giant Axonal 46 gene in the viral capsid n/a 9/27/2013 Neuropathy Hannah's Hope Fund 1‐(4‐benzhydrylpiperazin‐1‐yl)‐3,3‐ Management of postherpetic 47 diphenylpropan‐1‐one n/a 9/26/2013 neuralgia Zalicus Pharmaceuticals Ltd. Page 5 of 262 Orphan Drug Designations and Approvals List as of 12‐02‐2013 Governs January 1, 2014 ‐ March 31, 2014 6,8‐bis‐benzylsulfanyl‐octanoic Treatment of myelodysplastic 48 acid n/a 9/26/2013 syndrome Cornerstone Pharmaceuticals, Inc. Treatment of active Crohn's bacterium Bacteroides disease in the pediatric 49 thetaiotaomicron n/a 9/26/2013 population GT Biologics Ltd chimeric monoclonal antibody 50 against claudin‐18 splice variant 2 n/a 9/26/2013 Treatment of pancreatic cancer GANYMED Pharmaceuticals AG Treatment of adenoid cystic Novartis Pharmaceuticals 51 dovitinib n/a 9/26/2013 carcinoma Corporation Diagnostic for the management of patients with hepatocellular 52 ethiodized oil injection n/a 9/26/2013 carcinoma Guerbet LLC Treatment of follicular 53 idelalisib n/a 9/26/2013 lymphoma Gilead Sciences, Inc. Treatment of lymphoplasmacytic lymphoma with or without Walenstom's 54 idelalisib n/a 9/26/2013 macroglobulinemia Gilead Sciences, Inc. Use as a targeted contrast agent for diagnostic MRI for the detection and localization of focal liver lesions in patients manganese (II) chloride where gadolinium based tetrahydrate (with L‐alanine and contrast agents are vitamin D3 as promoters of contraindicated or cannot be 55 absorption) n/a 9/26/2013 administered CMC Contrast AB Treatment of patients with 56 melphalan hydrochloride n/a 9/26/2013 hepatocellular carcinoma Delcath Systems, Inc. small molecule inhibitor of Treatment of Huntington's 57 phosphodiesterase 10 n/a 9/26/2013 disease Omeros Corporation Page 6 of 262 Orphan Drug Designations and Approvals List as of 12‐02‐2013 Governs January 1, 2014 ‐ March 31, 2014 anti‐sense oligonucleotide consisting of 2ââ?¬â?¢0‐Me RNA with a phosphorothioate 58 backbone n/a 9/23/2013 Treatment of cystic fibrosis ProQR therapeutics B.V. Treatment of chronic thromboembolic pulmonary Bayer HealthCare Pharmaceuticals, 59 riociguat Adempas 9/19/2013 hypertension Inc. Treatment of pulmonary Bayer HealthCare Pharmaceuticals, 60 riociguat Adempas 9/19/2013 arterial hypertension. Inc. Treatment of follicular 61 lenalidomide Revlimid 9/13/2013 lymphoma Celgene Corporation 4‐[(5R)‐6,7‐Dihydro‐5H‐pyrrolo[1,2‐ c]imidazol‐5‐yl]‐3‐ Novartis Pharmaceuticals 62 flurorbenzonitrile n/a 9/13/2013 Treatment of Cushing's disease corporation anti‐CD3 mAb (SPV‐T3a)‐ricin A chain fusion protein and anti‐CD7 mAb (WT1)‐ricin A chain fusion Treatment of graft versus host 63 protein n/a 9/13/2013 disease Xenikos BV Treatment of human African melarsoprol‐ trypanosomiasis (sleeping Peter Kennedy, CBE, MD, PhD, DSc, 64 hydroxypropylbetadex n/a 9/13/2013 sickness) FRCP FMedSci, autologous ex vivo expanded CD4+‐ enriched leukocytes treated with the de‐methylating agent 5‐aza‐ Treatment of glioblastoma 65 2ââ?¬â?¢‐deoxycytidine Alecsat 9/13/2013 multiforme CytoVac A/S Treatment of patients with angioimmunoblastic T‐cell 66 brentuximab vedotin Adcetris 9/13/2013 lymphoma SeattleGenetics, Inc.
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  • 1 Therapeutic Antibodies – from Past to Future Stefan Dubel¨ and Janice M

    1 Therapeutic Antibodies – from Past to Future Stefan Dubel¨ and Janice M

    1 1 Therapeutic Antibodies – from Past to Future Stefan Dubel¨ and Janice M. Reichert 1.1 An Exciting Start – and a Long Trek In the late nineteenth century, the German army doctor Emil von Behring (1854–1917), later the first Nobel Laureate for Medicine, pioneered the thera- peutic application of antibodies. He used blood serum for the treatment of tetanus and diphtheria (‘‘Blutserumtherapie’’). When his data were published in 1890 [1], very little was known about the factors or mechanisms involved in immune defense. Despite this, his smart conclusion was that a human body needs some defense mechanism to fight foreign toxic substances and that these substances should be present in the blood – and therefore can be prepared from serum and used for therapy against toxins or infections. His idea worked, and the success allowed him to found the first ‘‘biotech’’ company devoted to antibody-based therapy in 1904 – using his Nobel Prize money as ‘‘venture capital.’’ The company is still active in the business today as part of CSLBehring. In 1908, Paul Ehrlich, the father of hematology [2] and the first consistent concept of immunology (‘‘lateral chain theory,’’ Figure 1.1d [3]), was awarded the second Nobel Prize related to antibody therapeutics for his groundbreaking work on serum, ‘‘particularly to the valency determination of sera preparations.’’ Ehrlich laid the foundations of antibody generation by performing systematic research on immunization schedules and their efficiency, and he was the first to describe different immunoglobulin subclasses. He also coined the phrases ‘‘passive vaccination’’ and ‘‘active vaccination.’’ His lateral chain theory (‘‘Seitenketten,’’ sometimes misleadingly translated to ‘‘side-chain theory’’) postulated chemical receptors produced by blood cells that fitted intruding toxins (antigens).