9/5/2018
`
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`List of Orphan Designations and Approvals
`
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`LES. Food and Drug Administrator:
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`
`List of Orphan Designations and Approvals
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`This list contains products that are currently designated as orphans by the Office of Orphan Products Development. Those designated products that are also
`
`approved for marketing will have a status 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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`  
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`To open a copy of this list as an Excel Spreadsheet, click here Qrphan Designated Products Spreadsheet
`
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`Generic Name: (R)—N—[2—(6—chloro—5—methoxy—lH—indol—3—yl)pr0pyl]acetamide Trade Name:
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`
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`
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`Orphan Indication: Treatment of circadian rhythm sleep disorders in blind people with no light perception
`
`Sponsor: Phase 2 Discovery, Inc. Contact: Dr. Frank Zemlan
`
`Address: Cincinnati, OH Phone: 5134756618 Fax:
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`
`Status: Designated Designation Date: 03—Oct—01 Marketing Approval Date:
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`
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`Trade Name:
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`Generic Name: l,5—(Butylimin0)—l,5 dideoxy,D—glucitol
`
`
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`Orphan Indication: Treatment of Fabry's disease.
`
`Sponsor: Oxford GlycoSciences Contact: Mr. Bruce Manning
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`
`Address: Oxfordshire OX14 3YS, UK Phone: (508) 393—3100 Fax: (508) 393—3780
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`
`Status: Designated Designation Date: l2—May—98 Marketing Approval Date:
`
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`Trade Name:
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`Generic Name: l,5—(Butylimin0)—l,5 dideoxy,D—glucitol
`
`
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`Orphan Indication: Treatment of Gaucher disease.
`
`Sponsor: Oxford GlycoSciences Contact: Mr. Bruce Manning
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`
`Address: Oxfordshire OX14 3YS, UK Phone: (508) 393—3100 Fax: (508) 393—3780
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`
`Status: Designated Designation Date: 29—May—98 Marketing Approval Date:
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`
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`methanesulfonate Trade Name:
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`Generic Name: l—( l l—dodecylamino— lO—hydroxyundecyl)—3 ,7—dimethylxanthine hydrogen
`
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`
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`
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`Orphan Indication: Treatment of hormone refractory prostate carcinoma.
`
`Sponsor: Cell Therapeutics, Inc. Contact: Ms. Jennie Allewell
`
`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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`
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`IGeneric Name: lllIndium pentetreotide Trade Name: SomatoTher
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`
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`Orphan Indication: Treatment of somatostatin receptor positive neuroendocrine tumors.
`
`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
`
`Status: Designated Designation Date: 10—Jun—99 Marketing Approval Date:
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`
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`IGeneric Name: 15AU81 ITrade Name: Remodulin
`
`
`
`
`Orphan Indication: Treatment of pulmonary arterial hypertension.
`
`Sponsor: United Therapeutics Corp. Contact: Mr. Dean Bunce
`
`
`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:
`
`
`
`Orphan Indication: Treatment of multiple myeloma.
`
`Sponsor: NeoRx Corporation Contact: Dr. Robert Caspari
`
`
`Address: Seattle, WA Phone: (206) 281—7001 Fax:
`
`
`Status: Designated Designation Date: 10—Feb—99 Marketing Approval Date:
`
`
`
`
`"Generic Name: 2'—3'—dideoxyadenosine l
`
`https://web.archive.org/web/20011120071654/http://www.fda.gov:80/orphan/designat/list.htm
`
`
`
`
`
`
`ALVOGEN, Exh. 1051, p. 0001
`
`1/125
`
`
`ALVOGEN, Exh. 1051, p. 0001
`
`

`

`9/5/2018
`
`
`ITrade Name:
`I
`
`
`Orphan Indication: Treatment of aquired immunodeficiency syndrome.
`
`Sponsor: National Cancer Institute, Dct Contact: Dr. Jay Greenblatt
`
`Address: Bethesda, MD Phone: (301) 496—7912 Fax:
`
`
`Status: Designated/Withdrawn Designation Date: 21—Jul—87 Marketing Approval Date:
`
`
`
`
`
`
`
`List of Orphan Designations and Approvals
`
`
`
`
`Generic Name: 2'—deoxycytidine
`
`Trade Name:
`
`
`
`Orphan Indication: As a host—protective agent in 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
`
`
`Status: Designated Designation Date: 09—Sep—96 Marketing Approval Date:
`
`
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`
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`
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` 
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`
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`IGeneric Name: 2—0—desulfated heparin Trade Name: Aeropin
`
`
`
`Orphan Indication: Treatment of cystic fibrosis.
`
`Sponsor: Kennedy & Hoidal, M.D.'s Contact: Dr. John Hoidal
`
`
`Address: Salt Lake City, UT Phone: (704) 355—2000 Fax:
`
`
`Status: Designated Designation Date: 17—Sep—93 Marketing Approval Date:
`
`
`
`
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`
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`
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`Trade Name:
`
`
`
`Generic Name: 2—chloroethyl—3—sarcosinamide—1—nitrosourea
`
`
`
`Orphan Indication: Treatment for malignant gliomas
`
`Sponsor: Lawrence Panasci, MD Contact: Dr. Lawrence Panasci
`
`
`Address: Montreal, Quebec H3T 1E2, Phone: 5143408248 Fax: 5143408302
`
`
`Status: Designated Designation Date: 03—Aug—01 Marketing Approval Date:
`
`
`
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`
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`
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`
`
`Generic Name: 2—methoxyestradiol Trade Name: Panzem
`
`
`
`Orphan Indication: Treatment of multiple myeloma
`
`Sponsor: EntreMed, Inc. Contact: Dr. Edward Gubish, Jr.,
`
`
`Address: Rockville, MD Phone: 3012179858 Fax: 3012179594
`
`
`Status: Designated Designation Date: 10—Jul—01 Marketing Approval Date:
`
`
`
`
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`
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`
`
`Generic Name: 24,25 dihydroxycholecalciferol
`
`Trade Name:
`
`
`
`Orphan Indication: 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
`
`
`
`
`Orphan Indication: 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:
`
`
`
`Orphan Indication: Treatment of Larnbert—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'—trii0d0thyroacetate
`
`Trade Name:
`
`
`
`Orphan Indication: Treatment of well—differentiated papillary, follicular or combined papillary/follicular
`
`carcinomas of the 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— 1 H—2ylmethylene)— 1 ,3—dihydro—indol—2—one
`
`
`https://web.archive.org/web/20011120071654/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
`
`
`
`ITrade Name:
`
`
`Orphan Indication: 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:
`
`
`
`
`
`
`
`I
`
`Generic Name: 3—(3 ,5—Dimethyl— lH—2ylmethylene)—l ,3—dihydro—indol—2—one Trade Name:
`
`
`
`
`
`
`Orphan Indication: Treatment of von Hippel—Lindau disease.
`
`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—l'—one)—l—piperidine—2,6—dione Trade Name: Revimid (proposed)
`
`
`
`
`Orphan Indication: 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:
`
`
`
`
`
`
` 
`
`
`
`ALVOGEN, Exh. 1051, p. 0003
`
`3/125
`
`
`Generic Name: 4—aminosalicylic acid Trade Name: Pamisyl (P—D), Rezipas (Squibb)
`
`
`
`
`Orphan Indication: 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:
`
`
`
`
`
`
`
`
`
`Generic Name: 4OSD02
`
`Trade Name:
`
`
`
`Orphan Indication: Treatment of chronic iron overload resulting from conventional transfusional treatment
`
`of beta—thalassemia major and sickle 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:
`
`
`
`
`
`
`
`Generic Name: 5,6—dihydro—5—azacytidine
`
`Trade Name:
`
`
`
`Orphan Indication: 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:
`
`
`
`
`
`
`
`Generic Name: 5—aza—2'—deoxycytidine
`
`Trade Name:
`
`
`
`Orphan Indication: Treatment of 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:
`
`
`
`
`
`
`
`
`
`Generic Name: 506U78
`
`Trade Name:
`
`
`
`Orphan Indication: Treatment of chronic lymphocytic leukemia.
`
`Sponsor: Glaxo Wellcome, Inc. Contact: Ms. Mama Doucette
`
`
`Address: Research Triangle Park, NC Phone: (919) 483—6222 Fax:
`
`
`Status: Designated Designation Date: 02—Sep—99 Marketing Approval Date:
`
`
`
` 
`
`
`
`Generic Name: 5a8, monoclonal antibody to CD4
`
`Trade Name:
`
`
`
`Orphan Indication: For use 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/20011120071654/http://www.fda.gov:80/orphan/designat/list.htm
`
`
`
`
`
`
`ALVOGEN, Exh. 1051, p. 0003
`
`

`

`List of Orphan Designations and Approvals
`
`
`9/5/2018
`
`Generic Name: 6—hydroxymethylacylfulvene
`
`Trade Name:
`
`
`
`Orphan Indication: Treatment of 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:
`
`
`
`
`
`Generic Name: 6—hydroxymethylacylfulvene
`
`Trade Name:
`
`
`
`Orphan Indication: Treatment of 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:
`
`
`
`
`
`
`
`
`
`Generic Name: 6—hydroxymethylacylfulvene
`
`Trade Name:
`
`
`
`Orphan Indication: 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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`
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`
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`
`
`Generic Name: 8 Cyclopentyl 1,3—dipr0pylxanthine
`
`Trade Name:
`
`
`Orphan Indication: Treatment of cystic 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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`
`
`IGeneric Name: 8—methoxsalen Trade Name: Uvadex
`
`
`
`
`Orphan Indication: For use in conjunction with the UVAR photopheresis 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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`
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`
`
`ALVOGEN, Exh. 1051, p. 0004
`
`4/ 125
`
`
`
`IGeneric Name: 8—methoxsalen Trade Name: Uvadex
`
`
`
`
`Orphan Indication: For the prevention of acute rejection of cardiac allografts.
`
`Sponsor: Therakos, Inc. Contact: Ms. Roberta Acchione
`
`Address: Exton, PA Phone: (610) 280—1008 Fax:
`
`
`Status: Designated Designation Date: l2—May—94 Marketing Approval Date:
`
`
`
`
`IGeneric Name: 9—cis retinoic acid Trade Name: Panretin
`
`
`
`
`Orphan Indication: 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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`
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`
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`
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`
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`Generic Name: 9—cis—retin0ic acid
`
`Trade Name:
`
`
`
`Orphan Indication: Prevention of retinal detachment due to 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:
`
`
`
`Orphan Indication: 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:
`
`
`
`
`
`
`
` 
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`|
`https://web.archive.org/web/20011120071654/http://www.fda.gov:80/orphan/designat/list.htm
`
`
`ALVOGEN, Exh. 1051, p. 0004
`
`

`

`List of Orphan Designations and Approvals
`9/5/2018
`
`
`
`Generic Name: 9—nitro—20—(S)—camptothecin||Trade Name: Camvirexl
`
`
`
`
`Orphan Indication: 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:
`
`
`
`Orphan Indication: 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:
`
`
`
`
`IGeneric Name: acetylcysteine Trade Name: Acetadote
`
`
`
`
`Orphan Indication: For the 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:
`
`
`
`
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`
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`
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`
`IGeneric Name: Acetylcysteine Trade Name: Mucomyst/Mucomyst 10 IV
`
`
`
`
`Orphan Indication: Intravenous treatment of 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:
`
`
`
`Orphan Indication: Treatment of 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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`IGeneric Name: Adeno—associated viral vector containing the gene for human coagulation factor IX ITrade Name: Coagulin—B
`
`
`
`Orphan Indication: 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:
`
`
`
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`
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`IGeneric Name: Adeno—associated viral vector containing the gene for human coagulation factor IX ITrade Name: Coagulin—B
`
`
`
`Orphan Indication: 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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`
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`
`
`
`
`Generic Name: Adeno—associated viral—based vector cystic fibrosis gene therapy
`
`Trade Name:
`
`
`Orphan Indication: Treatment of cystic 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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`
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`Generic Name: Adenosine
`
`Trade Name:
`
`
`
`Orphan Indication: For use in conjunction with BCNU in the treatment of 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:
`
`
`
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`
`IGeneric Name: Adenovirus—based vector Factor VIII complementary DNA to somatic cells Trade Name: MiniAdFVIII
`
`
`
`
`Orphan Indication: Treatment of hemophilia A.
`
`Sponsor: GenStar Therapeutics Corporation Contact: Dr. Carolyn Sidor
`
`
`
`https://web.archive.org/web/20011120071654/http://www.fda.gov:80/orphan/designat/list.htm
`
`
`5/ 125
`
`
`ALVOGEN, Exh. 1051, p. 0005
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`ALVOGEN, Exh. 1051, p. 0005
`
`

`

`List of Orphan Designations and Approvals
`9/5/2018
`
`
`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:
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`
`
`
`Orphan Indication: