Orphan Drugs: In Development

DEVELOPMENT
STATUS

DISEASE DRUG NAME
Cancer, Ovarian EthyolTM
(continued) amifostine

SPONSOR

FDA OFFICIAL DESIGNATION

U.S. Bioscience
(W. Conshohocken, PA)

use as a chemoprotective agent for
cyclophosphamide in the treatment
of advanced ovarian carcinoma

application submitted

OncoRad OV103 CYTOGEN treatment of ovarian cancer Phase I
Y + rium labeled (Princeton, NJ)
MAb
OncoScint OV103 CYTOGEN detection of ovarian cancer application submitted
indium IN 111 (Princeton, NJ)
murine MAb B72.3
taxol Bristol-Myers Squibb treatment of ovarian cancer Phase III
(New York, NY)
Cancer, PanorexTM Centocor treatment of pancreatic cancer Phase II
Pancreatic MAb 17-1A (Malvern, PA)
trimetrexate U.S. Bioscience treatment of pancreatic Phase III
glucuronate (West Conshohocken, PA) adenocarcinoma
Cancer, Renal Ampligen~ HEM Pharmaceutical treatment of renal cell carcinoma Phase II
(Philadelphia, PA)
Proleukin~ Cetus Oncology treatment of metastatic renal cell application submitted
interleukin-2 (Chiron) carcinoma
(recombinant) (Emeryville, CA)
Cancer, Ifex~ Bristol-Myers Squibb treatment of soft tissue sarcomas Phase II
Soft Tissue ifosfamide (New York, NY)
Cancer, Thyroid ThyrogenTM Genzyme adjunct in the diagnosis and Phase II
human thyroid (Cambridge, MA) treatment of thyroid cancer
stimulating factor
Triacana Medgenix Group for use in combination with levo- Phase I/II
tiratricol (Brussels, Belgium) thyroxine to suppress thyroid
stimulating hormone (TSH) in patients
with well-differentiated thyroid
cancer who are intolerant to adequate
doses of levo-thyroxine alone
Carnitine Carnitor~ Sigma-Tau treatment of manifestations of Phase III
Deficiency levocarnitine (Gaithersburg, MD) carnitine deficiency in patients with
end-stage renal disease (ESRD) who
require dialysis
Carnitor~ Sigma-Tau treatment of secondary carnitine application submitted
levocarnitine (Gaithersburg, MD) deficiency
Causalgia Ismelin~ I.V. CIBA-GEIGY treatment of moderate to severe application submitted
guanethidine (Summit, NJ) reflex sympathetic dystrophy and
monosulfate causalgia
Cerebral Palsy Oculinum~ Allergan treatment of dynamic muscle Phase II
botulinum toxin (Irvine, CA) contracture in pediatric cerebral
type A palsy patients
Cervical Dystonia Oculinum~ Allergan treatment of cervical dystonia application submitted
botulinum toxin (Irvine, CA)
type A
Charcot-Marie- dynamine Mayo Clinic treatment of hereditary motor and Phase II
Tooth Disease (Rochester, MN) sensory neuropathy type I
(Charcot-Marie-Tooth disease)
Chemotherapy sucralfate suspension Naska Pharmacal treatment of oral complications of Phase III
Complications (Lincolnton, NC) chemotherapy in bone marrow
transplant patients
Zinecard Adria for the prevention of Phase III
dexrazoxane for (Columbus, OH) cardiomyopathy associated with
injection doxorubicin administration
Cirrhosis Actigall CIBA-GEIGY management of the clinical signs Phase III

ursodeoxycholic acid

(Summit, NJ)

v 7
and symptoms associated with
primary biliary cirrhosis

Ursofalk
ursodeoxycholic acid

Interfalk
(Plattsburgh, NY)

treatment of patients with primary
biliary cirrhosis

Phase III