Orphan Drugs: In Development

DEVELOPMENT
DISEASE DRUG NAME SPONSOR FDA OFFICIAL DESIGNATION STATUS
Cytomegalovirus cytomegalovirus Miles Inc. for use in conjunction with Phase III
(CMV) immune globulin (Berkeley, CA) ganciclovir sodium for the treatment
intravenous (human) of CMV pneumonia in bone marrow
transplantation patients
SDZ MSL-109 Sandoz treatment of CMV retinitis in Phase II
(East Hanover, NJ) patients with AIDS
SDZ MSL-109 Sandoz prophylaxis of CMV disease in Phase II
(East Hanover, NJ) patients undergoing solid organ
transplantation
Dermatitis sulfapyridine Jacobus Pharmaceutical treatment of dermatitis Phase III
Herpetiformis (Princeton, NJ) herpetiformis
Diarrhea bovine ImmuCell Corp. treatment of diarrhea in AIDS Phase I
anti-cryptosporidium (Portland, ME) patients caused by infection with
immunoglobulin Cryptosporidium parvum
(BACI)
Drug Overdose Mucomyst Apothecon intravenous treatment of patients Phase III
acetylcysteine (Princeton, NJ) presenting with moderate to severe
acetaminophen overdose
Dysphagia sucralfate suspension Naska Pharmacal treatment of oral ulcerations and Phase III
(Lincolnton, NC) dysphagia in patients with
epidermolysis bullosa
Dysplasia, recombinant human BioTechnology General for the prevention of broncho- Phase I
Broncho- superoxide (New York, NY) pulmonary dysplasia in premature
pulmonary dismutase infants weighing less than 1,500
grams
Epilepsy antiepilepsirine Children's Hospital treatment of drug resistant application submitted
(Columbus, OH) generalized tonic-clonic (GTC)
epilepsy in children and adults
fosphenytoin Warner-Lambert acute treatment of patients with Phase III
(Morris Plains, NJ) status epilepticus of the grand mal
type
Esophageal Glypressin~ Ferring Laboratories treatment of bleeding esophageal Phase III
Varices, terlipressin (Suffern, NY) varices
Bleeding Sotradecol~ Elkins-Sinn treatment of bleeding esophageal Phase III
sodium tetradecyl (Cherry Hill, NJ) varices
sulfate
Fabry's Disease FABRase Robert Desnick, M.D. treatment of Fabry's disease Phase I/II
alpha-galactosidase A Mount Sinai School of
Medicine
(New York, NY)
Fistulas, Zecnil~ Ferring Laboratories adjunct to the non-operative Phase III
Cutaneous somatostatin (Suffern, NY) management of secreting cutaneous
fistulas of the stomach, duodenum,
small intestine (jejunum and ileum)
or pancreas
Gaucher's recombinant beta- Genzyme for replacement therapy in patients Phase III
Disease glucocerebrosidase (Cambridge, MA) with types I, II, and III Gaucher's
disease
Glycoside Digidote Boehringer Mannheim treatment of life-threatening acute application submitted
Intoxication digoxin immune Fab (Rockville, MD) cardiac glycoside intoxication
(ovine) manifested by conduction disorders,
ectopic ventricular activity and (in
some cases) hyperkalemia
Graft Rejection interleukin-1 alpha, Immunex for the promotion of early Phase II
human (Seattle, WA) engraftment in bone marrow

(recombinant) transplantation
Sandimmune Sandoz treatment of patients at high risk of Phase II
cyclosporine 2% (East Hanover, NJ) graft rejection following penetrating
ophthalmic ointment keratoplasty
Graft Vs. Host Orthozyme~ CD5 + Xoma for in-vivo treatment of bone marrow Phase II
Disease CD5-T lymphocyte (Berkeley, CA) recipients to prevent graft rejection
(GVHD) immunotoxin and graft vs. host disease (GVHD)
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