Orphan Drugs: In Development

DISEASE DRUG NAME
Angioedema C 1-inhibitor (human)
(continued) vapor heated

SPONSOR
Immuno Clinical
Research
(New York, NY)

FDA OFFICIAL DESIGNATION
treatment of acute attacks of
angioedema

DEVELOPMENT
STATUS
Phase III

Anorexia Marinol Unimed for the stimulation of appetite and Phase III
dronabinol (Somerville, NJ) prevention of weight loss in patients
with a confirmed diagnosis of AIDS
Megace~ Bristol-Myers Squibb treatment of anorexia, cachexia, or Phase III
megestrol acetate (New York, NY) significant weight loss (> 10% of
baseline body weight) and confirmed
diagnosis of AIDS
OxandrinTM Gynex Pharmaceuticals adjunctive therapy for AIDS patients Phase II
oxandrolone (Vernon Hills, IL) suffering from HIV wasting syndrome
Saizen~ Serono Laboratories treatment of AIDS-associated Phase II
somatropin for (Norwell, MA) catabolism/weight loss
injection
Bacteremia HA-l1ATM Centocor treatment of patients with gram- application submitted
nebacumab (Malvern, PA) negative bacteremia which has
progressed to endotoxin shock
Blepharospasm DysportTM Porton Products Ltd. treatment of essential blepharospasm Phase II/III
clostridium botulinum (Maidenhead, England)
toxin type A
Botulism, Infant botulism immune California Department treatment of infant botulism Phase II/III
globulin (human) of Health Services
(Berkeley, CA)
Burns epidermal growth Ethicon promotion of cutaneous wound Phase II
factor (human) (Somerville, NJ) healing in extreme burn treatment
protocols
Leucomax~ Schering-Plough treatment of severe thermal injuries Phase III
granulocyte/ (Madison, NJ) in patients with greater than 40%
macrophage-colony full or partial thickness burns
stimulating factor
RheothRx~ Burroughs Wellcome treatment of severe burns requiring Phase I
poloxamer 188 (Rsch. Triangle Park, NC) hospitalization
Cachexia Megace~ Bristol-Myers Squibb (See Anorexia)
megestrol acetate (New York, NY)
Cancer, Adrenal gossypol Marcus Reidenberg, M.D. treatment of cancer of the adrenal Phase II
(New York, NY) cortex
Cancer, Bladder INTRON~ A Schering-Plough treatment of carcinoma in situ of Phase III
interferon alfa-2b (Madison, NJ) the urinary bladder
(recombinant)
Photofrin~ QLT Phototherapeutics photodynamic therapy in patients Phase III
profimer sodium (Pearl River, NY) with transitional cell carcinoma in
situ of urinary bladder
Cancer, Bone Ifex~ Bristol-Myers Squibb treatment of bone sarcomas Phase II
ifosfamide (New York, NY)
Isovorin Lederle for use in conjunction with high- application submitted
l-leucovorin (Wayne, NJ) dose methotrexate in the treatment
of osteosarcoma
Cancer, Brain adenosine Medco Research treatment of brain tumors in Phase I
(Los Angeles, CA) conjunction with BCNU
Cancer, Breast toremifene Adria hormonal therapy of metastatic Phase III
(Columbus, OH) carcinoma of the breast
Cancer, Cervical Mitolactol~ Amswiss Scientific treatment of recurrent invasive Phase III
dibromodulcitol (New York, NY) metastatic squamous carcinoma of
the cervix
Cancer, Colorectal 5-fluorouracil Hoffmann-La Roche for use in combination with Phase III
(5-FU) (Nutley, NJ) Roferon~-A for the treatment of
advanced colorectal carcinoma
Immther Immuno Therapeutics treatment of pulmonary and hepatic Phase II
disaccharide tripeptide (Moorhead, MN) metastases in patients with
glycerol dipalmitoyl colorectal adenocarcinoma