Orphan Drugs: In Development

DEVELOPMENT
DISEASE DRUG NAME SPONSOR FDA OFFICIAL DESIGNATION STATUS
Cognitive EctapramTM Cambridge NeuroScience for the management of cognitive Phase III
Impairment pramiracetam sulfate (Cambridge, MA) dysfunction and enhancement of
antidepressant activity associated
with electroconvulsive therapy
Colitis Altracin A.L. Laboratories antibiotic-associated pseudo- Phase III
bacitracin (Fort Lee, NJ) membranous enterocolitis caused by
toxins A and B elaborated by
clostridium difficile
Pamisyl (P-D) Warren Beeken, M.D. treatment of mild to moderate Phase III
4-aminosalicylic acid Univ. of Vermont ulcerative colitis in patients
(Burlington, VT) intolerant of sulfasalazine
short-chain fatty acid Richard Breuer, M.D. treatment of the active phase of Phase II
solution (Evanston, IL) ulcerative colitis with involvement
restricted to the left side of the
colon
Corneal Disease DehydrexTM Holles Laboratories treatment of recurrent corneal Phase III
(Cohasset, MA) erosion unresponsive to
conventional therapy
epidermal growth Chiron IntraOptics acceleration of corneal epithelial Phase III
factor (human) (Irvine, CA) regeneration and healing of stromal
incisions from corneal transplant
surgery
fibronectin (human The New York treatment of non-healing corneal Phase III
plasma derived) Blood Center ulcers or epithelial defects which
(New York, NY) have been unresponsive to conventional therapy and the underlying
cause has been eliminated
ofloxacin solution Allergan treatment of bacterial corneal ulcers Phase II
(Irvine, CA)
Cushing's Acthrel Ferring Laboratories differentiating pituitary and ectopic application submitted
Syndrome ovine corticotropin (Suffern, NY) production of ACTH in patients
releasing hormone with ACTH-dependent Cushing's
syndrome
Cryptococcal amphotericin B Bristol-Myers Squibb treatment of cryptococcal meningitis Phase II
Meningitis lipid complex (New York, NY)
Cyanide hydroxycobalamin/ Everka, Inc. treatment of severe acute cyanide Phase II
Poisoning sodium thiosulfate (San Francisco, CA) poisoning
Cystic Fibrosis amiloride HCL Glaxo treatment of cystic fibrosis Phase III
solution for (Rsch. Triangle Park, NC)
inhalation
DNase Genentech to reduce mucous viscosity and Phase III
(S. San Francisco, CA) enable the clearance of airway
secretions in patients with cystic
fibrosis
recombinant human Synergen treatment of cystic fibrosis Phase I/II
secretory leucocyte (Boulder, CO)
protease inhibitor
Uendex Thomas Kennedy, M.D. adjunct to the treatment of cystic Phase I
dextran sulfate and John Hoidal, M.D. fibrosis
(Richmond, VA)
Cystinosis cysteamine HCL Warner-Lambert treatment of nephropathic Phase I
(Morris Plains, NJ) cystinosis
phosphocysteamine Medea Research treatment of cystinosis Phase III
Laboratories
(Port Jefferson

Station, NY)
Cystitis, ELMIRON~ Baker Cummins treatment of interstitial cystitis application submitted
Interstitial pentosan (Miami, FL)
polysulphate sodium
nifedipine Jonathan Fleischmann, treatment of interstitial cystitis Phase III
M.D.
(Cleveland, OH)
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