LIST OF ORPHAN PRODUCTS DESIGNATIONS AND APPROVALS ThroughDecember 31,1999 NAME SPONSOR &. ADDRESS Generic Name DD=Date Designated TN=Trade Name INDICATION DESIGNATED MA=Marketing Approval 1,S-(Butylimino)- 1,5 Treatment of Fabry’s disease. Oxford GlycoSciences dideoxy,D-glucitol 10, The Quadrant TN= Abington Science Park, Abington Oxfordshire OX14 3YS UK, DD=05/12/1998 MA= I / 1,5-(Butylimino)- 1,5 Treatment of Gaucher disease. Oxford GlycoSciences dideoxy,D-glucitol 10, The Quadrant TN= Abington Science Park, Abington Oxfordshire OX14 3YS UK, DD=05/29/1998 MA= / / 11Hndium pentetreotide Treatment of somatostatinreceptor positive neuroendocrine Louisiana State University Medical Center TN= SomatoTher tumors. Foundation 1600 Canal St. 10th Floor New Orleans, LA 70112 DD=06/10/1999 MA= / / 15AU81 Treatment of pulmonary arterial hypertension. United Therapeutics Corp. TN= 68 T.W. Alexander Dr. P.O. Box 14186 ResearchTriangle Park, NC 27709 DD=06/04/1997 MA= ! 1 166Ho-DOTMP Treatment of multiple myeloma. NeoRx Corporation TN= 4 10 W. Harrison Seattle, WA 98119 DD=02/10/1999 MA= I / 2’-deoxycytidine As a host-protective agent in the treatment of acute Grant, Steven M.D. TN= myelogenous leukemia. Massey Cancer Center, VCU P.O. Box 980230 Richmond, VA 23298 DD=O9/09/1996 MA= ! I 2-0-desulfated heparin Treatment of cystic fibrosis. Kennedy & Hoidal, M.D.‘s TN= Aeropin University of Utah Health SciencesCenter 50 North Medical Drive, Room 4R240 Salt Lake City, UT 84132 DD=09/17/1993 MA= / / 24,25 dihydroxycholecalciferol Treatment of uremic osteodystrophy. Lemrnon Company TN= 1510 Delp Drive Kulpsville, PA 19443 DD=02/27/1987 MA= I I 3:4-diaminopyridine Treatment of Lambert-Eaton myasthenic syndrome. JacobusPharmaceutical Company TN= 37 Cleveland Avenue P.O. Box 5290 Princeton, NJ 08540 DD=12/18/1990 MA= / / Page 1 LIST OF ORPHAN PRODUCTS DESIGNATIONS AND APPROVALS ThroughDecember 3 1,1999 NAME SPONSOR & ADDRESS Generic Name DD=Date Designated TN=Trade Name INDICATION DESIGNATED MA=varketing Approval 3-(3,5-dimethyl-lH-2ylmethyle Treatment of Kaposi’s sarcoma. Sugen, Inc. ne)- 1,3-dihydro-indol-2-one 230 East Grand Ave. TN= South San Francisco, CA 94080 DD=09/11/1998 MA= / / 4-aminosalicylic acid Treatment of mild to moderateulcerative colitis in patients Beeken, Warren M.D. TN= Pamisyl (P-D), Rezipas intolerant to sulfasalazine. University Of Vermont (Squibb) Given C-3 17 Burlington, VT 05405 DD=I2/13/1989 MA= / / 40SD02 Treatment of chronic iron overload resulting from conventional Biomedical Frontiers, Inc. TN= transfusional treatment of beta-thalassemiamajor and sickle cell 1095 10th Ave., SE. anemia. Minneapolis, MN 55414 DD=12/21/1998 MA= I / 5,6-dihydro-5-azacytidine Treatment of malignant mesothelioma. ILEX Oncology, Inc. TN= 11550 IH 10 West, Suite 300 San Antonio, TX 78230 DD=05/11/1992 MA= / I 5-aza-2’-deoxycytidine Treatment of acute leukemia. PharmachemieU.S.A., Inc. TN= 1510 Delp Dr. Kulpsville, PA 19443 DD=08/03/1987 MA= / 1 506U78 Treatment of chronic lyrnphocytic leukemia. Glaxo Wellcome, Inc. TN= Five Moore Dr. P.O. Box 13398 Durham, NC 27709 DD=09/02/1999 MA= I I 5a8, monoclonal antibody to For use in post-exposureprophylaxis for occupational exposure Biogen, Inc. CD4 to human immunodeficiency virus. 14 Cambridge Center TN= Cambridge, MA 02142 DD=12/20/1993 MA= I f 6-hydroxymethylacylfulvene Treatment of histologically confirmed advancedor metastatic MGI Pharrna, Inc. TN= pancreatic cancer. 6300 West Old ShakoppeRd. Suite 110 Bloomington, MN 55438 DD=04/06/1999 MA= ! / 6-hydroxymethylacylfulvene Treatment of ovarian cancer. MGI Pharrna, Inc. TN= 6300 West Old ShakoppeRd. Suite 110 Bloomington, MN 55438 DD=07/06/1999 MA= I I 6-hydroxymethylacylfulvene Treatment of renal cell carcinoma. MGI Pharma, Inc. TN= 6300 West Old ShakoppeRd. Suite 110 Bloomington, MN 55438 DD=07/27/1999 MA= I I Page 2 LIST OF ORPHAN PRODUCTS DESIGNATIONS AND APPROVALS ThroughDecember 3 1,1999 NAME SPONSOR & ADDRESS Generic Name DD=Date Designated INDICATION DESIGNATED TN=Trade Name MA=Marketing Approval 8 Cyclopentyl Treatment of cystic fibrosis. SciClone Pharmaceuticals,Inc. 1,3-dipropylxanthine 901 Mariner’s Island Boulevard TN= San Mateo, CA 94404 DD=03/24/1997 MA= I I 8-methoxsalen For use in conjunction with the WAR photopheresisto treat Therakos, Inc. TN= Uvadex diffuse systemic sclerosis. Oaklands Corporate Center 437 Creamery Way Exton, PA 19341 DD=06/22/1993 MA= / / 8-methoxsalen For the prevention of acute rejection of cardiac allografts. Therakos, Inc. TN= Uvadex Oaklands Corporate Center 437 Creamery Way Exton, PA 19341 DD=O5/12/1994 MA= / I 9-cis retinoic acid Treatment of acute promyelocytic leukemia. Ligand Pharmaceuticals,Inc. TN= Panretin 9393 Towne Centre Drive Suite 100 San Diego, CA 92121 DD=04/10/1992 MA= / / 9-nitro-20-(S)-camptothecin Treatment of pancreatic cancer. Stehlin Foundation for Cancer Research TN= 1315 St. JosephParkway, Suite 1818 Houston, TX 77002 DD=O9/16/1996 MA= / I API903 Treatment of acute graft-versus-host diseasein patients Ariad Gene Therapeutics, Inc. TN= undergoing bone marrow transplantation. 26 Landsdowne St. Cambridge, MA 02139 DD=11/24/1999 MA= / / APL 400-020 V-Beta DNA Treatment of cutaneousT cell lymphoma. Wyeth-Lederle Vaccines and Pediatrics vaccine 211 Bailey Rd. TN= West Henrietta, NY 14586 DD=03/08/1995 MA= I / Acetylcysteine Intravenous treatment of patients presenting with moderateto Apothecon TN= MucomystMucomyst 10 severe acetaminophenoverdose. P.O. Box 4500 IV Princeton, NJ 08543 DD=08/13/1987 MA= I I Aconiazide Treatment of tuberculosis. Lincoln Diagnostics TN= P.O. Box 1128 Decatur, IL 62525 DD=06/20/1988 MA= I I Adeno-associatedviral-based Treatment of cystic fibrosis. Targeted Genetics Corporation vector cystic fibrosis gene 1100 Olive Way, Suite 100 therapy Seattle, WA 98101 TN= DD=02/15/1995 MA= / I Page 3 LIST OF ORPHAN PRODUCTS DESIGNATIONS AND APPROVALS ThroughDecember 3 1,1999 NAME SPONSOR & ADDRESS Generic Name DD=Date Designated TN=Trade Name INDICATION DESIGNATED MA=Marketing Approval Adenovirus-based vector Factor Treatment of hemophilia A. GenStar Therapeutics Corporation VIII complementary DNA to 10835 Altman Row somatic cells Suite 150 TN= MiniAdFVIII San Diego, CA 92121 DD=12/15/1999 MA= I / Albendazole Treatment of hydatid disease(cystic echinococcosisdue to E. SmithKline Beecham Pharmaceuticals TN= Albenza granulosus larvae or alveolar echinococcosisdue to E. One Franklin Plaza multilocularis larvae). P.O. Box 7929 Philadelphia, PA 1910 1 DD=01/17/1996 MA=06/11/1996 Albendazole Treatment of neurocysticercosis due to Taenia solium as: 1) SmithKline Beecham Pharmaceuticals TN= Albenza chemotherapy of parenchymal, subarachnoidal and racemose One Franklin Plaza (cysts in spinal fluid) neurocysticercosis in symptomatic cases P.O. Box 7929 and 2) prophylaxis of epilepsy and other sequelaein Philadelphia, PA 19101 asymptomatic neurocysticercosis. DD=O1/18/1996 MA=0611 111996 Aldesleukin Treatment of primary immunodeficiency diseaseassociated with Chiron Corporation TN= Proleukin T-cell defects. 4560 Horton Street Emeryville, CA 94608 DD=03/22/1989 MA= I / Aldesleukin Treatment of metastatic renal cell carcinoma. Chiron Corporation TN= Proleukin 4560 Horton Street Emeryville, CA 94608 DD=09/14/1988 MA=0510511992 Aldesleukin Treatment of metastaticmelanoma. Chiron Corporation TN= Proleukin 4560 Horton Street Emeryville, CA 94608 DD=09/10/1996 MA=O1/09/1998 Aldesleukin Treatment of acute myelogenous leukemia. Chiron Corporation TN= Proleukin 4560 Horton St. Emeryville, CA 94608 DD=07/31/1998 MA= / I Aldesleukin For the treatment non-Hodgkin’s lyrnphoma. Chiron Corporation TN= Proleukin 4560 Horton St. Emeryville, CA 94608 DD=11/24/1998 MA= I I Alglucerase injection For replacement therapy in patients with Gaucher’sdisease type Genzyme Corporation TN= Ceredase I. One Kendall Square Cambridge, MA 02139 DD=03/11/1985 MA=04/05/1991 Alglucerase injection Replacementtherapy in patients with Type II and III Gaucher’s Genzyme Corporation TN= Ceredase disease. One Kendall Square Cambridge, MA 02 139 DD=07/21/1995 MA= I I Page 4 LIST OF ORPHAN PRODUCTS DESIGNATIONS AND APPROVALS ThroughDecember 3 1, 1999 NAME SPONSOR & ADDRESS Generic Name DD=Date Designated TN=Trade Name INDICATION DESIGNATED MA=Marketing ., Approval Alitretinoin Topical treatment of cutaneouslesions in patients with Ligand PharmaceuticalsInc. TN= Panretin AIDS-related Kaposi’s sarcoma. 10275 Science Center Drive San Diego, CA 92121 DD=03/24/1998 MA=02/02/1999 Allogeneic peripheral blood Treatment of pancreatic cancer. Applied Immunotherapeutics, LLC mononuclear cells sensitized 14132 E. Firestone Boulevard against patient alloantigens by Santa Fe Springs, CA 90670 mixed lymphocyte culture DD=OG/13/1997 MA= / I TN= CYTOIMPLANT Allopurinol sodium Management of patients with leukemia, lymphoma, and solid Catalytica Pharmaceuticals,Inc TN= Zyloprim for Injection tumor malignancies who are receiving cancer therapy which PO Box 1887 causeselevations of serum and urinary uric acid levels and who Greenville, NC 27835 cannot tolerate oral therapy. DD=10/16/1992 MA=05/17/1996 Alpha-l -antitrypsin As supplementation therapy for alpha-l-antitrypsin deficiency in Chiron Corporation (recombinant DNA Origin) the ZZ phenotype population. 4560 Horton Street TN= Emeryville, CA 94608 DD=01/01/1984 MA= / / Alpha-galactosidaseA Treatment of Fabry’s disease. Desnick, Robert J. M.D. TN= Fabrase The Mount Sinai School Of Medicine Fifth Avenue at 100th Street, Box 1203 New York, NY 10029 DD=07/20/1990 MA= I I Alpha-galactosidaseA