9/5/2018
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`List of Orphan Designations and Approvals
`
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`U.S. Food and Drug Administration
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`List of Orphan Designations and Approvals
`
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`This list contains productsthat are currently designated as orphans by the Office of Orphan Products Development. Those designated products that are also
`
`approved for marketing will haveastatus listing of "Designated/Approved"as well as a marketing approval date. To search for a particular product, please use the
`
`Find option of your Web browser(generally this is found under the Edit menu)
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`To open a copyofthis list as an Excel Spreadsheet, click here Orphan Designated Products Spreadsheet
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`Generic Name: (R)-N-[2-(6-chloro-5-methoxy-1H-indol-3-yl)propyl]acetamide Trade Name:
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`
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`OrphanIndication: Treatment of circadian rhythm sleep disordersin blind people with nolight perception
`
`Sponsor: Phase 2 Discovery, Inc. Contact: Dr. Frank Zemlan
`
`Address: Cincinnati, OH Phone: 5134756618 Fax:
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`
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`Status: Designated Designation Date: 03-Oct-01 Marketing Approval Date:
`
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`Trade Name:
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`Generic Name: 1,5-(Butylimino)-1,5 dideoxy,D-glucitol
`
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`OrphanIndication: Treatment of Fabry's disease.
`
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`Sponsor: Oxford GlycoSciences Contact: Mr. Bruce Manning
`
`Address: Oxfordshire OX14 3YS, UK Phone: (508) 393-3100 Fax: (508) 393-3780
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`
`Status: Designated Designation Date: 12-May-98 Marketing Approval Date:
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`methanesulfonate Trade Name:
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`Generic Name: 1-(11-dodecylamino-10-hydroxyundecy])-3,7-dimethylxanthine hydrogen
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`OrphanIndication: Treatment of hormonerefractory prostate carcinoma.
`
`Sponsor: Cell Therapeutics, Inc. Contact: Ms. Jennie Allewell
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`Address: Seattle, WA Phone: (206) 282-7100 Fax:
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`Status: Designated Designation Date: 18-Jan-00 Marketing Approval Date:
`
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`ALVOGEN, Exh. 1051, p. 0001
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`[Generic Name:111Indium pentetreotide||Trade Name: SomatoTher
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`OrphanIndication: Treatment of somatostatin receptor positive neuroendocrine tumors.
`
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`Sponsor: Louisiana State University Medical Center Foundation Contact: Mr. James Hardy
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`Address: New Orleans, LA Phone: (504) 568-3712 Fax: (504) 568-3460
`
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`Status: Designated Designation Date: 10-Jun-99 Marketing Approval Date:
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`[Generic Name: 15AU81 [Trade Name: Remodulin
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`OrphanIndication: Treatment of pulmonary arterial hypertension.
`
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`Sponsor: United Therapeutics Corp. Contact: Mr. Dean Bunce
`
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`Address: Research Triangle Park, NC Phone: (919) 485-8352 Fax: (919) 485-8352
`
`Status: Designated Designation Date: 04-Jun-97 Marketing Approval Date:
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`Generic Name: 166Ho-DOTMP
`
`Trade Name:
`
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`OrphanIndication: Treatment of multiple myeloma.
`
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`Sponsor: NeoRx Corporation Contact: Dr. Robert Caspari
`
`Address: Seattle, WA Phone: (206) 281-7001 Fax:
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`
`
`Status: Designated Designation Date: 10-Feb-99 Marketing Approval Date:
`
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`|Generic Name:2'-3'-dideoxyadenosine |
`https://web.archive.org/web/200 1112007 1654/http://www.fda.gov:80/orphan/designat/list.-htm
`
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`
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`Trade Name:
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`Generic Name: 1,5-(Butylimino)-1,5 dideoxy,D-glucitol
`
`
`
`OrphanIndication: Treatment of Gaucherdisease.
`
`
`Sponsor: Oxford GlycoSciences Contact: Mr. Bruce Manning
`
`Address: Oxfordshire OX14 3YS, UK Phone: (508) 393-3100 Fax: (508) 393-3780
`
`
`Status: Designated Designation Date: 29-May-98 Marketing Approval Date:
`
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`ALVOGEN, Exh. 1051, p. 0001
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`

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`9/5/2018
`
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`[Trade Name:
`|
`
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`OrphanIndication: Treatment of aquired immunodeficiency syndrome.
`
`Sponsor: National CancerInstitute, Dct Contact: Dr. Jay Greenblatt
`
`Address: Bethesda, MD Phone: (301) 496-7912 Fax:
`
`
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`Status: Designated/Withdrawn Designation Date: 21-Jul-87 Marketing Approval Date:
`
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`List of Orphan Designations and Approvals
`
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`Generic Name: 2'-deoxycytidine
`
`Trade Name:
`
`
`OrphanIndication: Asa host-protective agentin the treatment of acute myelogenous leukemia.
`
`Sponsor: Grant, Steven M.D. Contact: Dr. Steven Grant
`
`Address: Richmond, VA Phone: (804) 828-5122 Fax: (804) 828-8079
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`
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`Status: Designated Designation Date: 09-Sep-96 Marketing Approval Date:
`
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`[Generic Name: 2-0-desulfated heparin|/Trade Name: Aeropin
`
`
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`OrphanIndication: Treatmentofcystic fibrosis.
`
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`Sponsor: Kennedy & Hoidal, M.D.'s Contact: Dr. John Hoidal
`
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`Address: Salt Lake City, UT Phone: (704) 355-2000 Fax:
`
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`Status: Designated Designation Date: 17-Sep-93 Marketing Approval Date:
`
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`Trade Name:
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`Generic Name: 2-chloroethy|-3-sarcosinamide- 1 -nitrosourea
`
`
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`OrphanIndication: Treatment for malignant gliomas
`
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`Sponsor: Lawrence Panasci, MD Contact: Dr. Lawrence Panasci
`
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`Address: Montreal, Quebec H3T 1E2, Phone: 5143408248 Fax: 5143408302
`
`Status: Designated Designation Date: 03-Aug-01 Marketing Approval Date:
`
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`Generic Name: 2-methoxyestradiol|/Trade Name: Panzem
`
`
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`OrphanIndication: Treatment of multiple myeloma
`
`Sponsor: EntreMed, Inc. Contact: Dr. Edward Gubish,Jr.,
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`
`Address: Rockville, MD Phone: 3012179858 Fax: 3012179594
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`
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`Status: Designated Designation Date: 10-Jul-01 Marketing Approval Date:
`
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`Generic Name: 24,25 dihydroxycholecalciferol
`
`Trade Name:
`
`
`OrphanIndication: Treatment of uremic osteodystrophy.
`
`
`Sponsor: Lemmon Company Contact: Ms. Deborah Jaskot
`
`
`Address: Kulpsville, PA Phone: (215) 256-8400 Fax:
`
`
`Status: Designated Designation Date: 27-Feb-87 Marketing Approval Date:
`
`
`
`Generic Name: 3'-azido-2' ,3'dideoxyuridine|/Trade Name: AZDU
`
`
`
`
`OrphanIndication: Treatment of acquired immunodeficiency syndrome.
`
`Sponsor: Berlex Laboratories, Inc. Contact: Donald Gay
`
`Address: Alameda, CA Phone: (510) 769-4985 Fax:
`
`
`
`Status: Designated/Withdrawn Designation Date: 20-Nov-89 Marketing Approval Date:
`
`
`
`
`
`
` 
`
`
`
`
`
`Generic Name: 3 ,4-diaminopyridine
`
`Trade Name:
`
`
`OrphanIndication: Treatment of Lambert-Eaton myasthenic syndrome.
`
`Sponsor: Jacobus Pharmaceutical Company Contact: Dr. Neil Lewis
`
`Address: Princeton, NJ Phone: (609) 921-7447 Fax:
`
`
`
`Status: Designated Designation Date: 18-Dec-90 Marketing Approval Date:
`
`
`
`
`
`
`
`
`Generic Name:3,5 ,3'-triiodothyroacetate
`
`Trade Name:
`
`
`OrphanIndication: Treatment of well-differentiated papillary, follicular or combined papillary/follicular
`
`carcinomasofthe thyroid gland.
`
`
`Sponsor: Elliot Danforth, Jr., M.D. Contact: Dr. Elliot Danforth, Jr.
`
`Address: Underhill, VT Phone: 8028992349 Fax: 8028992349
`
`
`
`Status: Designated Designation Date: 20-Sep-00 Marketing Approval Date:
`
`
`
`
`
`
`
`
`Generic Name: 3-(3 5-dimethyl-1H-2ylmethylene)- 1 ,3-dihydro-indol-2-one
`
`
`
`
`https://web.archive.org/web/200 1112007 1654/http://www.fda.gov:80/orphan/designat/list.-htm
`
`
`
`
`ALVOGEN, Exh. 1051, p. 0002
`
`2/125
`
`
`ALVOGEN, Exh. 1051, p. 0002
`
`

`

`List of Orphan Designations and Approvals
`9/5/2018
`
`
`
`[Trade Name:
`|
`
`
`OrphanIndication: Treatment of Kaposi's sarcoma.
`
`Sponsor: Sugen, Inc. Contact: Dr. Stephen Carter
`
`Address: South San Francisco, CA Phone: (650) 553-8300 Fax: (650) 553-8301
`
`
`Status: Designated Designation Date: 11-Sep-98 Marketing Approval Date:
`
`
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`
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`
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`Generic Name: 3-(3 5-Dimethy]-1H-2ylmethylene)-1 ,3-dihydro-indol-2-one Trade Name:
`
`
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`
`
`
`OrphanIndication: Treatment of von Hippel-Lindaudisease.
`
`
`Sponsor: Sugen, Inc. Contact: Dr. Paul Scigalla
`
`Address: South San Francisco, CA Phone: (650) 553-8792 Fax: (650) 553-8314
`
`
`Status: Designated Designation Date: 23-Mar-00 Marketing Approval Date:
`
`
`
`
`Generic Name: 3-(4'aminoisoindoline-1'-one)- 1-piperidine-2 ,6-dione|/Trade Name: Revimid (proposed)
`
`
`
`
`OrphanIndication: Treatment for multiple myeloma
`
`
`Sponsor: Celegene Corporation Contact: Dr. Steve Thomas
`
`Address: Warren, NJ Phone: 7328053914 Fax: 7322714184
`
`
`
`Status: Designated Designation Date: 20-Sep-01 Marketing Approval Date:
`
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`
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` 
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`Generic Name: 4-aminosalicylic acid|/Trade Name: Pamisyl (P-D), Rezipas (Squibb)
`
`
`
`
`OrphanIndication: Treatment of mild to moderate ulcerative colitis in patients intolerant to sulfasalazine.
`
`Sponsor: Beeken, Warren M.D. Contact: Dr. Warren Beeken
`
`
`Address: Burlington, VT Phone: (802) 656-2554 Fax:
`
`
`Status: Designated Designation Date: 13-Dec-89 Marketing Approval Date:
`
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`
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`
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`
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`
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`
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`Generic Name: 40SD02
`
`Trade Name:
`
`
`OrphanIndication: Treatment of chronic iron overload resulting from conventional transfusional treatment
`
`of beta-thalassemia majorandsickle cell anemia.
`
`
`Sponsor: Biomedical Frontiers, Inc. Contact: Dr. Paul Dragsten
`
`
`Address: Minneapolis, MN Phone: (612) 378-0228 Fax: 6123783601
`
`
`Status: Designated Designation Date: 21-Dec-98 Marketing Approval Date:
`
`
`
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`
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`
`
`Generic Name: 5,6-dihydro-5-azacytidine
`
`Trade Name:
`
`
`OrphanIndication: Treatment of malignant mesothelioma.
`
`
`Sponsor: ILEX Oncology, Inc. Contact: Mr. Edward Martinez
`
`Address: San Antonio, TX Phone: (210) 949-8270 Fax: (210) 949-8282
`
`
`
`Status: Designated Designation Date: 11-May-92 Marketing Approval Date:
`
`
`
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`
`
`Generic Name: 5-aza-2'-deoxycytidine
`
`Trade Name:
`
`
`OrphanIndication: Treatmentof acute leukemia.
`
`
`Sponsor: SuperGen, Inc. Contact: Dr. Sam Boddapati
`
`Address: Dublin, CA Phone: 9255600100 Fax: 9255516472
`
`
`
`Status: Designated Designation Date: 03-Aug-87 Marketing Approval Date:
`
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`
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`Generic Name: 506U78
`
`Trade Name:
`
`
`OrphanIndication: Treatment of chronic lymphocytic leukemia.
`
`
`Sponsor: Glaxo Wellcome, Inc. Contact: Ms. Marna Doucette
`
`
`Address: Research Triangle Park, NC Phone: (919) 483-6222 Fax:
`
`
`Status: Designated Designation Date: 02-Sep-99 Marketing Approval Date:
`
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` 
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`Generic Name: 5a8, monoclonal antibody to CD4
`
`Trade Name:
`
`
`OrphanIndication: Foruse in post-exposure prophylaxis for occupational exposure to human
`
`immunodeficiency virus.
`
`
`Sponsor: Biogen, Inc. Contact: Ms. Sylvie Gregoire
`
`
`Address: Cambridge, MA Phone: (617) 679-2631 Fax: (617) 679-3170
`
`
`Status: Designated/Withdrawn Designation Date: 20-Dec-93 Marketing Approval Date:
`
`
`
`https://web.archive.org/web/200 1112007 1654/http://www.fda.gov:80/orphan/designat/list.-htm
`
`
`
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`
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`ALVOGEN, Exh. 1051, p. 0003
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`3/125
`
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`ALVOGEN, Exh. 1051, p. 0003
`
`

`

`9/5/2018
`
`
`List of Orphan Designations and Approvals
`
`
`
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`Generic Name: 6-hydroxymethylacylfulvene
`
`Trade Name:
`
`
`OrphanIndication: Treatmentof ovarian cancer.
`
`Sponsor: MGI Pharma, Inc. Contact: Dr. Jo Gustafson
`
`
`
`Address: Bloomington, MN Phone: 9523464722 Fax: 9523464800
`
`
`Status: Designated Designation Date: 06-Jul-99 Marketing Approval Date:
`
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`
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`Generic Name: 6-hydroxymethylacylfulvene
`
`Trade Name:
`
`
`OrphanIndication: Treatmentof renal cell carcinoma.
`
`Sponsor: MGI Pharma, Inc. Contact: Dr. Jo Gustafson
`
`
`
`Address: Bloomington, MN Phone: 9523464722 Fax: (612) 346-4800
`
`
`Status: Designated Designation Date: 27-Jul-99 Marketing Approval Date:
`
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`
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`
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`
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`Generic Name: 6-hydroxymethylacylfulvene
`
`Trade Name:
`
`
`OrphanIndication: Treatment of histologically confirmed advanced or metastatic pancreatic cancer.
`
`Sponsor: MGI Pharma, Inc. Contact: Dr. Jo Gustafson
`
`
`Address: Bloomington, MN Phone: 9523464722 Fax: 9523464800
`
`
`Status: Designated Designation Date: 06-Apr-99 Marketing Approval Date:
`
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`Generic Name: 8 Cyclopenty] 1 ,3-dipropylxanthine
`
`Trade Name:
`
`
`OrphanIndication: Treatmentofcystic fibrosis.
`
`
`Sponsor: SciClone Pharmaceuticals, Inc. Contact: Mr. David Wilgus
`
`Address: San Mateo, CA Phone: 6503583456 Fax: (415) 358-3469
`
`
`
`Status: Designated Designation Date: 24-Mar-97 Marketing Approval Date:
`
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`
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` 
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`[Generic Name: 8-methoxsalen|/Trade Name: Uvadex
`
`
`
`
`OrphanIndication: For use in conjunction with the UVARphotopheresis to treat diffuse systemic
`
`sclerosis.
`
`
`Sponsor: Therakos, Inc. Contact: Ms. Roberta Acchione
`
`Address: Exton, PA Phone: (610) 280-1008 Fax:
`
`
`
`Status: Designated Designation Date: 22-Jun-93 Marketing Approval Date:
`
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`[Generic Name: 8-methoxsalen|/Trade Name: Uvadex
`
`
`
`
`OrphanIndication: Forthe prevention ofacute rejection of cardiac allografts.
`
`Sponsor: Therakos, Inc. Contact: Ms. Roberta Acchione
`
`Address: Exton, PA Phone: (610) 280-1008 Fax:
`
`
`
`Status: Designated Designation Date: 12-May-94 Marketing Approval Date:
`
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`
`[Generic Name:9-cis retinoic acid||/Trade Name:Panretin
`
`
`
`
`OrphanIndication: Treatment of acute promyelocytic leukemia.
`
`
`Sponsor: Ligand Pharmaceuticals, Inc. Contact: Dr. Howard Holden
`
`
`Address: San Diego, CA Phone: (619) 550-7600 Fax: (619) 625-9524
`
`
`Status: Designated Designation Date: 10-Apr-92 Marketing Approval Date:
`
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`Generic Name: 9-cis-retinoic acid
`
`Trade Name:
`
`
`OrphanIndication: Prevention of retinal detachmentdueto proliferative vitreoretinopathy.
`
`Sponsor: Allergan, Inc. Contact: Dr. Trudy Rumbaugh
`
`Address: Irvine, CA Phone: (714) 246-4292 Fax: (714) 246-4272
`
`
`
`Status: Designated/Withdrawn Designation Date: 02-Jan-97 Marketing Approval Date:
`
`
`
`
`
`
`
`
`Generic Name: 9-nitro-20-(S)-camptothecin
`
`Trade Name:
`
`
`OrphanIndication: Treatment of pancreatic cancer.
`
`
`Sponsor: Stehlin Foundation for Cancer Research Contact: Dr. Beppino Giovanella
`
`Address: Houston, TX Phone: (713) 659-1336 Fax: (713) 659-1503
`
`
`Status: Designated Designation Date: 16-Sep-96 Marketing Approval Date:
`
`
`
`
`
`
`
`
` 
`
`a||
`ALVOGEN, Exh. 1051, p. 0004
`4/125
`https://web.archive.org/web/200 1112007 1654/http://www.fda.gov:80/orphan/designat/list.-htm
`
`
`
`ALVOGEN, Exh. 1051, p. 0004
`
`

`

`9/5/2018
`List of Orphan Designations and Approvals
`
`
`Generic Name: 9-nitro-20-(S)-camptothecin||Trade Name: Camvirex|
`
`
`
`
`OrphanIndication: Treatment of pediatric HIV infection/AIDS
`
`Sponsor: NovoMed Pharmaceuticals, Inc. Contact: Dr. M. Reza Sadaie
`
`
`Address: Germantown, MD Phone: 3012756625 Fax: 5093525349
`
`
`Status: Designated Designation Date: 15-May-01 Marketing Approval Date:
`
`
`
`
`
`
`Generic Name: Abetimus
`
`Trade Name:
`
`
`
`OrphanIndication: Treatment of lupus nephritis.
`
`Sponsor: La Jolla Pharmaceutical Co. Contact: Dr. Andrew Wiseman
`
`
`
`Address: San Diego, CA Phone: (858) 452-6600 Fax:
`
`Status: Designated Designation Date: 28-Jul-00 Marketing Approval Date:
`
`
`
`
`
`[Generic Name:acetylcysteine|/Trade Name: Acetadote’
`
`
`
`
`OrphanIndication: Forthe intravenous treatment of moderate to severe acetaminophen overdose
`
`Sponsor: Cumberland Pharmaceuticals Inc. Contact: Mr. Peter Vaccari
`
`Address: Nashville, TN Phone: 3013091260 Fax: 3013098470
`
`
`Status: Designated Designation Date: 19-Oct-01 Marketing Approval Date:
`
`
`
`
`
`
`
`[Generic Name: Acetylcysteine|/Trade Name: Mucomyst/Mucomyst10 IV
`
`
`
`
`OrphanIndication: Intravenous treatmentof patients presenting with moderate to severe acetaminophen
`
`overdose.
`
`
`
`Sponsor: Bristol-Myers Squibb Company Contact: Ms. Barbara LoPiccolo
`
`Address: Princeton, NJ Phone: 6098972470 Fax: (609) 897-5515
`
`
`
`
`Status: Designated Designation Date: 13-Aug-87 Marketing Approval Date:
`
`
`
`
`
`
`
`Generic Name: Aconiazide
`
`Trade Name:
`
`
`OrphanIndication: Treatmentof tuberculosis.
`
`
`Sponsor: Lincoln Diagnostics Contact: Mr. Gary Hein
`
`Address: Decatur, IL Phone: (800) 537-1336 Fax:
`
`
`
`Status: Designated Designation Date: 20-Jun-88 Marketing Approval Date:
`
`
`
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` 
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`
`[Generic Name: Adeno-associated viral vector containing the gene for human coagulation factor IX [Trade Name: Coagulin-B
`
`
`
`
`OrphanIndication: Intrahepatic treatment of patients with moderate to severe hemophilia
`
`Sponsor: Avigen, Inc. Contact: Ms. Janice Castillo
`
`Address: Alameda, CA Phone: 5107487282 Fax:
`
`
`Status: Designated Designation Date: 13-Jun-01 Marketing Approval Date:
`
`
`
`
`
`[Generic Name: Adeno-associated viral vector containing the gene for human coagulation factor IX [Trade Name: Coagulin-B
`
`
`
`
`OrphanIndication: Intramuscular treatment of patients with moderate to severe hemophilia
`
`Sponsor: Avigen, Inc. Contact: Ms. Janice Castillo
`
`Address: Alameda, CA Phone: 5107487150 Fax: 5107487155
`
`
`
`
`Status: Designated Designation Date: 13-Jun-01 Marketing Approval Date:
`
`
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`
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`
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`
`
`Generic Name: Adeno-associated viral-based vectorcystic fibrosis gene therapy
`
`Trade Name:
`
`
`OrphanIndication: Treatmentofcystic fibrosis.
`
`
`Sponsor: Targeted Genetics Corporation Contact: Mr. Barry Polenz
`
`Address: Seattle, WA Phone: (206) 521-7818 Fax: (206) 223-0288
`
`
`
`
`Status: Designated Designation Date: 15-Feb-95 Marketing Approval Date:
`
`
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`
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`
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`
`
`
`
`Generic Name: Adenosine
`
`Trade Name:
`
`
`OrphanIndication: Foruse in conjunction with BCNUinthe treatmentof brain tumors.
`
`Sponsor: Medco Research,Inc. Contact: Dr. Sam Teichman
`
`
`Address: Los Angeles, CA Phone: (213) 966-4155 Fax:
`
`
`
`Status: Designated/Withdrawn Designation Date: 01-Aug-89 Marketing Approval Date:
`
`
`
`
`[Generic Name: Adenovirus-based vector Factor VIII complementary DNAto somaticcells |[Trade Name: MiniAdFVIII
`
`
`
`
`OrphanIndication: Treatment of hemophilia A.
`
`
`Sponsor: GenStar Therapeutics Corporation Contact: Dr. Carolyn Sidor
`
`
`https://web.archive.org/web/200 1112007 1654/http://www.fda.gov:80/orphan/designat/list.htm
`
`
`5/125
`
`
`ALVOGEN, Exh. 1051, p. 0005
`
`ALVOGEN, Exh. 1051, p. 0005
`
`

`

`9/5/2018
`
`
`List of Orphan Designations and Approvals
`
`
`Address: San Diego, CA Phone: (919) 361-2286 Fax: (919) 361-2290
`
`
`Status: Designated Designation Date: 15-Dec-99 Marketing Approval Date:
`
`
`
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` 
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`Generic Name: adenovirus-mediated herpes simplex virus-thymidine kinase gene Trade Name:
`
`
`
`
`OrphanIndication: Use with gancyclovir in the treatment of malign