Orphan Drugs: In Development

DEVELOPMENT
DISEASE DRUG NAME SPONSOR FDA OFFICIAL DESIGNATION STATUS
Spasticity, Neuralgon Mericon Industries treatment of intractable spasticity Phase II
Intractable L-baclofen (Peoria, IL) associated with spinal cord injury
(continued) or multiple sclerosis
Thrombocythemia Agrelin~ Roberts Pharmaceutical treatment of essential Phase III
anagrelide (Eatontown, NJ) thrombocythemia (ET)
Thrombo- Arvin~ Knoll Pharmaceuticals antithrombotic for patients with Phase II
cytopenia ancrod (Whippany, NJ) heparin-induced thrombocytopenia
or thrombosis who require immediate
and continued anticoagulation
iloprost Berlex treatment of heparin-associated application submitted
(Wayne, NJ) thrombocytopenia
Tissue/Organ Nizoral Pharmedic for use with cyclosporine A to Phase I
Transplantation ketaconazole (Wheeling, IL) diminish the nephrotoxicity induced
by cyclosporine in organ
transplantation
Trigeminal Neuralgon Gerhard Fromm, M.D. treatment of trigeminal neuralgia Phase II
Neuralgia L-baclofen (Pittsburgh, PA)
Tuberculosis aconiazide Lincoln Diagnostics treatment of tuberculosis Phase II
(Decatur, IL)
Rifater~ Marion Merrell Dow short-course treatment of Phase III
rifampin, isoniazid, (Kansas City, MO) tuberculosis
pyrazinamide
Turner's ethinyl estradiol - Gynex Pharmaceuticals treatment of Turner's syndrome Phase III S
Syndrome (Vernon Hills, IL)
Humatrope~ Eli Lilly treatment of short stature associated in clinical trials
somatropin of (Indianapolis, IN) with Turner's syndrome
recombinant DNA
origin
Norditropin~ Novo-Nordisk treatment of short stature associated Phase III
somatropin (Princeton, NJ) with Turner's syndrome
Nutropin Genentech treatment of short stature associated Phase III
somatropin for (S. San Francisco, CA) with Turner's syndrome
injection
OxandrinTM Gynex Pharmaceuticals treatment of short stature associated Phase III
oxandrolene (Vernon Hills, IL) with Turner's syndrome
Uremic 24, 25 dihydroxy- TAG Pharmaceuticals/ treatment of uremic osteodystrophy Phase III
Osteodystrophy cholecalciferol Lemmon Company
(Sellersville, PA)
Ventricular bethanidine sulfate Medco Research treatment of primary ventricular Phase III
Fibrillation (Los Angeles, CA) fibrillation
bethanidine sulfate Medco Research prevention of recurrence of primary Phase II/III
(Los Angeles, CA) ventricular fibrillation
Ventricular Betapace~ Bristol-Myers Squibb prevention of life-threatening application submitted
Tachyarrhythmias d,l-sotalol HCL (New York, NY) ventricular tachyarrhythmias
Betapace~ Bristol-Myers Squibb treatment of life-threatening application submitted
d,l-sotalol HCL (New York, NY) ventricular tachyarrhythmias
Von Willebrand's DDAVP Rhone-Poulenc Rorer (see Hemophilia)
Disease desmopressin acetate (Collegeville, PA)
NovoSevenTM Novo-Nordisk A/S (See Hemophilia)
factor VIIa (Copenhagen, Denmark)
(recombinant DNA
origin)
Vulvar VulvanTM Star Pharmaceuticals treatment of vulvar dystrophies Phase III completed
Dystrophies testosterone (Pompano Beach, FL)
propionate ointment
2%

Wilson's Disease zinc acetate Lemmon Company treatment of Wilson's disease Phase III
(Sellersville, PA)
Xerostomia Salagen~ Tablets MGI PHARMA treatment of xerostomia induced by application submitted
pilocarpine HCL (Minneapolis, MN) radiation therapy for head and neck
cancer