Orphan Drug Designations and Approvals List as of 12‐01‐2014

309 Pages·2014·1.74 MB·English

Orphan Drug Designations and Approvals List as of 12‐01‐2014
Governs January 1, 2015 ‐ March 31, 2015
Row Contact
Generic Name Trade Name Designation Date Designation
Num Company/Sponsor
1 Altretamine Hexalen 2/9/1984 Treatment of advanced Medimmune
adenocarcinoma of the ovary. Oncology, Inc.
2 Levocarnitine Carnitor 2/28/1984 Treatment of genetic carnitine Sigma-Tau
deficiency. Pharmaceuticals, Inc.
3 Pentamidine isethionate Pentam 300 2/28/1984 Treatment of Pneumocystis Fujisawa USA, Inc.
carinii pneumonia.
4 Cromolyn sodium Gastrocrom 3/8/1984 Treatment of mastocytosis. Fisons Corporation
5 Bacitracin Altracin 3/13/1984 Treatment of antibiotic- A. L. Laboratories, Inc.
associated pseudomembranous
enterocolitis caused by toxins A
and B elaborated by Clostridium
difficile.
6 Hemin Panhematin 3/16/1984 Amelioration of recurrent attacks Abbott Laboratories
of acute intermittent porphyria
(AIP) temporarily related to the
menstrual cycle in susceptible
women and similar symptoms
which occur in other patients
with AIP, porphyria variegata and
hereditary coproporphyria.
7 Botulinum toxin type A Botox 3/22/1984 Treatment of strabismus and Allergan, Inc.
blepharospasms
8 Ethanolamine oleate Ethamolin 3/22/1984 Treatment of patients with QOL Medical
esophageal varices that have
recently bled, to prevent
rebleeding.
9 Succimer Chemet Capsules 5/9/1984 Treatment of lead poisoning in Bock Pharmacal
children. Company
Page 1 of 309
Orphan Drug Designations and Approvals List as of 12‐01‐2014
Governs January 1, 2015 ‐ March 31, 2015
Row Contact
Generic Name Trade Name Designation Date Designation
Num Company/Sponsor
10 Pegademase bovine Adagen 5/29/1984 For enzyme replacement therapy Sigma-tau
for ADA deficiency in patients Pharmaceuticals, Inc.
with severe combined
immunodeficiency.
11 Monooctanoin Moctanin 5/30/1984 For dissolution of cholesterol Ethitek
gallstones retained in the Pharmaceuticals, Inc.
common bile duct.
12 Clofazimine Lamprene 6/11/1984 Treatment of lepromatous Novartis
leprosy, including dapsone- Pharmaceutical
resistant lepromatous leprosy Corporation
and lepromatous leprosy
complicated by erythema
nodosum leprosum.
13 Levocarnitine Carnitor 7/26/1984 Treatment of primary and Sigma-Tau
secondary carnitine deficiency of Pharmaceuticals, Inc.
genetic origin.
14 Iodine 131 6B-iodomethyl-19- n/a 8/1/1984 For use in adrenal cortical David E. Kuhl, M.D.
norcholesterol imaging.
15 Chenodiol Chenix 9/21/1984 For patients with radiolucent Solvay
stones in well opacifying
gallbladders, in whom elective
surgery would be undertaken
except for the presence of
increased surgical risk due to
systemic disease or age.
16 Pentamidine isethionate n/a 10/29/1984 Treatment of Pneumocystis Aventis Behring L.L.C.
carinii pneumonia.
17 L-5 Hydroxytrytophan n/a 11/1/1984 Treatment of postanoxic Watson Laboratories,
intention myoclonus. Inc.
Page 2 of 309
Orphan Drug Designations and Approvals List as of 12‐01‐2014
Governs January 1, 2015 ‐ March 31, 2015
Row Contact
Generic Name Trade Name Designation Date Designation
Num Company/Sponsor
18 Urogastrone n/a 11/1/1984 For acceleration of corneal Chiron Vision
epithelial regeneration and
healing of stromal incisions from
corneal transplant surgery.
19 Digoxin immune FAB (Ovine) Digibind 11/1/1984 Treatment of potentially life Glaxo Wellcome Inc.
threatening digitalis intoxication
in patients who are refractory to
management by conventional
therapy.
20 Potassium citrate Urocit-K 11/1/1984 For avoidance of the University of Texas
complication of calcium stone Health Science Center
formation in patients with uric at Dallas
lithiasis.
21 Potassium citrate Urocit-K 11/1/1984 Prevention of calcium renal University of Texas
stones in patients with Health Science Center
hypocitraturia. at Dallas
22 Potassium citrate Urocit-K 11/1/1984 Prevention of uric acid University of Texas
nephrolithiasis. Health Science Center
at Dallas
23 Teniposide Vumon For Injection 11/1/1984 Treatment of refractory Bristol-Myers Squibb
childhood acute lymphocytic Pharmaceutical
leukemia. Research Institute
24 Selegiline HCl Eldepryl 11/7/1984 As an adjuvant to levodopa and Somerset
carbidopa treatment of Pharmaceuticals, Inc.
idiopathic Parkinson's disease
(paralysis agitans),
postencephalitic Parkinsonism,
and symptomatic Parkinsonism.
Page 3 of 309
Orphan Drug Designations and Approvals List as of 12‐01‐2014
Governs January 1, 2015 ‐ March 31, 2015
Row Contact
Generic Name Trade Name Designation Date Designation
Num Company/Sponsor
25 Antithrombin III (human) Thrombate Iii 11/26/1984 For replacement therapy in Bayer Corporation
congenital deficiency of AT-III for
prevention and treatment of
thrombosis and pulmonary
emboli.
26 Alpha1-proteinase inhibitor Prolastin 12/7/1984 For replacement therapy in the Bayer Corporation
(human) alpha-1-proteinase inhibitor
congenital deficiency state.
27 Trientine HCl Syprine 12/24/1984 Treatment of patients with Merck Sharp &
Wilson's disease who are Dohme Research
intolerant, or inadequately
responsive to penicillamine.
28 factor XIII concentrate Corifact 1/16/1985 Treatment of congenital factor CSL Behring LLC
(human) XIII deficiency
29 Levomethadyl acetate Orlaam 1/24/1985 Treatment of heroin addicts Biodevelopment
hydrochloride suitable for maintenance on Corporation
opiate agonists.
30 Antithrombin III (human) Atnativ 2/8/1985 For the treatment of patients Pharmacia & Upjohn
with hereditary antithrombin III AB
deficiency in connection with
surgical or obstetrical procedures
or when they suffer from
thromboembolism.
Page 4 of 309
Orphan Drug Designations and Approvals List as of 12‐01‐2014
Governs January 1, 2015 ‐ March 31, 2015
Row Contact
Generic Name Trade Name Designation Date Designation
Num Company/Sponsor
31 Superoxide dismutase (human) n/a 3/6/1985 For protection of donor organ Pharmacia-Chiron
tissue from damage or injury Partnership
mediated by oxygen-derived free
radicals that are generated
during the necessary periods of
ischemia (hypoxia, anoxia), and
especially reperfusion, associated
with the operative procedure.
32 Digoxin immune fab(ovine) Digidote 3/11/1985 Treatment of life-threatening Boehringer
acute cardiac glycoside Mannheim Corp.
intoxication manifested by
conduction disorders, ectopic
ventricular activity and (in some
cases) hyperkalemia.
33 Alglucerase injection Ceredase 3/11/1985 For replacement therapy in Genzyme Corporation
patients with Gaucher's disease
type I.
34 Naltrexone HCl Trexan 3/11/1985 For blockade of the DuPont
pharmacological effects of Pharmaceuticals
exogenously administered
opioids as an adjunct to the
maintenance of the opioid-free
state in detoxified formerly
opioid-dependent individuals.
35 Oxymorphone Numorphan H.P. 3/19/1985 For relief of severe intractable DuPont Merck
pain in narcotic-tolerant patients. Pharmaceutical
Company
Page 5 of 309
Orphan Drug Designations and Approvals List as of 12‐01‐2014
Governs January 1, 2015 ‐ March 31, 2015
Row Contact
Generic Name Trade Name Designation Date Designation
Num Company/Sponsor
36 Tretinoin n/a 4/15/1985 Treatment of squamous Hannan Ophthalmic
metaplasia of the ocular surface Marketing Services,
epithelia (conjunctiva and/or Inc
cornea) with mucous deficiency
and keratinization.
37 Surface active extract of saline Infasurf 6/7/1985 Treatment and prevention of ONY, Inc.
lavage of bovine lungs respiratory failure due to
pulmonary surfactant deficiency
in preterm infants.
38 Anagrelide Agrylin 6/11/1985 Treatment of polycythemia vera. Roberts
Pharmaceutical Corp.
39 Midodrine HCl Amatine 6/21/1985 Treatment of patients with Schier Ridgewood
symptomatic orthostatic F.K.A. (Roberts
hypotension. Pharmaceutical Corp.)
40 Defibrotide n/a 7/5/1985 Treatment of thrombotic Crinos International
thrombocytopenic purpura.
41 Zidovudine Retrovir 7/17/1985 Treatment of AIDS related Glaxo Wellcome Inc.
complex.
42 zidovudine Retrovir 7/17/1985 Treatment of AIDS Glaxo Wellcome Inc.
43 Cromolyn sodium 4% Opticrom 4% Ophthalmic 7/24/1985 Treatment of vernal Fisons Corporation
ophthalmic solution Solution keratoconjunctivitis.
44 Ifosfamide Ifex 8/7/1985 Treatment of soft tissue Bristol-Myers Squibb
sarcomas. Pharmaceutical
Research Institute
45 Ifosfamide Ifex 8/7/1985 Treatment of bone sarcomas Bristol-Myers Squibb
Company
Page 6 of 309
Orphan Drug Designations and Approvals List as of 12‐01‐2014
Governs January 1, 2015 ‐ March 31, 2015
Row Contact
Generic Name Trade Name Designation Date Designation
Num Company/Sponsor
46 Pentosan polysulfate sodium Elmiron 8/7/1985 Treatment of interstitial cystitis. Alza Corporation
47 Pentastarch Pentaspan 8/28/1985 As an adjunct in leukapheresis to Du Pont
improve the harvesting and Pharmaceuticals
increase the yield of leukocytes
by centrifugal means.
48 Rifampin, isoniazid, Rifater 9/12/1985 For the short-course treatment Hoechst Marion
pyrazinamide of tuberculosis. Roussel
49 Epoprostenol Flolan 9/25/1985 Treatment of primary pulmonary Glaxo Wellcome Inc.
hypertension.
50 Methotrexate sodium Methotrexate 10/21/1985 Treatment of osteogenic Lederle Laboratories
sarcoma.
51 Zinc acetate Galzin 11/6/1985 Treatment of Wilson's disease. Lemmon Company
52 Mesna Mesnex 11/14/1985 For use as a prophylactic agent in Degussa Corporation
reducing the incidence of
ifosfamide-induced hemorrhagic
cystitis.
53 Somatrem for injection Protropin 12/9/1985 Treatment of short stature Genentech, Inc.
associated with Turner's
syndrome.
54 Rifampin Rifadin I.V. 12/9/1985 For antituberculosis treatment Hoechst Marion
where use of the oral form of the Roussel
drug is not feasible.
55 Somatrem for injection Protropin 12/9/1985 For long-term treatment of Genentech, Inc.
children who have growth failure
due to a lack of adequate
endogenous growth hormone
secretion.
Page 7 of 309
Orphan Drug Designations and Approvals List as of 12‐01‐2014
Governs January 1, 2015 ‐ March 31, 2015
Row Contact
Generic Name Trade Name Designation Date Designation
Num Company/Sponsor
56 Antithrombin III human Antithrombin Iii Human 1/2/1986 Preventing or arresting episodes American National
of thrombosis in patients with Red Cross
congenital AT-III deficiency
and/or to prevent the occurrence
of thrombosis in patients with AT-
III deficiency who have
undergone trauma or who are
about to undergo surgery or
parturition.
57 Flunarizine Sibelium 1/6/1986 Treatment of alternating Janssen Research
hemiplegia. Foundation
58 Tiopronin Thiola 1/17/1986 Prevention of cystine Pak, Charles Y.C. M.D.
nephrolithiasis in patients with
homozygous cystinuria.
59 Benzoate and phenylacetate Ucephan 1/21/1986 For adjunctive therapy in the ImmunexImmunex
prevention and treatment of
hyperammonemia in patients
with urea cycle enzymopathy due
to carbamylphosphate
synthetase, ornithine,
transcarbamylase, or
argininosuccinate synthetase
deficiency.
60 Beractant Survanta Intratracheal 2/5/1986 Treatment of neonatal Ross Laboratories
Suspension respiratory distress syndrome.
61 Beractant Survanta Intratracheal 2/5/1986 Prevention of neonatal Ross Laboratories
Suspension respiratory distress syndrome.
Page 8 of 309
Orphan Drug Designations and Approvals List as of 12‐01‐2014
Governs January 1, 2015 ‐ March 31, 2015
Row Contact
Generic Name Trade Name Designation Date Designation
Num Company/Sponsor
62 Terlipressin Glypressin 3/6/1986 Treatment of bleeding Ferring Laboratories,
esophageal varices. Inc.
63 Etidronate disodium Didronel 3/21/1986 Treatment of hypercalcemia of MGI Pharma, Inc.
malignancy inadequately
managed by dietary modification
and/or oral hydration.
64 Diethyldithiocarbamate Imuthiol 4/3/1986 Treatment of AIDS. Connaught
Laboratories
65 Epoetin alfa Epogen 4/10/1986 Treatment of anemia associated Amgen, Inc.
with end stage renal disease.
66 Eflornithine HCl Ornidyl 4/23/1986 Treatment of Trypanosoma Hoechst Marion
brucei gambiense infection Roussel
(sleeping sickness).
67 Cysteamine n/a 5/1/1986 Treatment of nephropathic Thoene, Jess G., M.D.
cystinosis.
68 Trimetrexate glucuronate Neutrexin 5/15/1986 Treatment of Pneumocystis Medimmune
carinii pneumonia in AIDS Oncology, Inc.
patients.
69 Sodium tetradecyl sulfate Sotradecol 6/10/1986 Treatment of bleeding Elkins-Sinn, Inc.
esophageal varices.
70 Somatropin for injection Humatrope 6/12/1986 For the long-term treatment of Eli Lilly and Company
children who have growth failure
due to inadequate secretion of
normal endogenous growth
hormone.
71 Anagrelide Agrylin 7/14/1986 Treatment of thrombocytosis in Roberts
chronic myelogenous leukemia. Pharmaceutical Corp.
Page 9 of 309
Orphan Drug Designations and Approvals List as of 12‐01‐2014
Governs January 1, 2015 ‐ March 31, 2015
Row Contact
Generic Name Trade Name Designation Date Designation
Num Company/Sponsor
72 Erwinia L-asparaginase Erwinase 7/30/1986 Treatment of acute lymphocytic Jazz Pharmaceuticals,
leukemia. Inc.
73 Chlorhexidine gluconate Peridex 8/18/1986 For use in the amelioration of Procter & Gamble
mouthrinse oral mucositis associated with Company
cytoreductive therapy used in
conditioning patients for bone
marrow transplantation therapy.
74 Botulinum toxin type A Botox 8/20/1986 Treatment of cervical dystonia. Allergan, Inc.
75 Nebacumab Centoxin 10/1/1986 Treatment of patients with gram- Centocor, Inc.
negative bacteremia which has
progressed to endotoxin shock.
76 Mazindol Sanorex 12/8/1986 Treatment of Duchenne muscular Collipp, Platon J. M.D.
dystrophy.
77 Leucovorin Leucovorin Calcium 12/8/1986 For use in combination with 5- Immunex Corporation
fluorouracil for the treatment of
metastatic colorectal cancer.
78 Zalcitabine n/a 12/9/1986 Treatment of AIDS. National Cancer
Institute, DCT
79 Teriparatide Parathar 1/9/1987 Diagnostic agent to assist in Rhone-Poulenc Rorer
establishing the diagnosis in Pharmaceuticals, Inc.
patients presenting with clinical
and laboratory evidence of
hypocalcemia due to either
hypoparathyroidism or
pseudohypoparathyroidism.
Page 10 of 309