Orphan Drugs: In Development

DISEASE
Cancer, Ovarian

DRUG NAME
Hexalen~
altretamine

COMPANY INDICATION
U.S. Bioscience treatment of advanced adenocarcinoma of the
(W Conshohocken, PA) ovary

Cancer, Testicular Ifex~ Bristol-Myers Squibb treatment of testicular cancer
Ifosfamide (New York, NY)
Carnitine Deficiency Carnitor~ Sigma-Tau treatment of primary carnitine deficiency of
levocarnitine (Gaithersburg, MD) genetic origin
Vita Carn~ McGaw, Inc. treatment of genetic carnitine deficiency
levocarnitine (Irvine, CA)
Coma Triostat~ SmithKline Beecham treatment of myxedema coma/precoma
liothyronine sodium (Philadelphia, PA)
injection
Cystitis, Hemorrhagic Mesnex~ Degussa Corp. prophylactic to reduce the incidence of
mesna (S. Hackensack, NJ) ifosfamide-induced hemorrhagic cystitis
Cytomegalovirus cytomegalovirus Mass. Public Health prevention or attenuation of primary CMV in
(CMV) immune globulin Biologic Labs immunosuppressed recipients of organ transplants
(human) (Jamaica Plain, MA)
Cytovene~ Syntex treatment of CMV retinitis in immunocompromised
ganciclovir sodium (Palo Alto, CA) patients, including people with AIDS
Digitalis Intoxication Digibind~ Burroughs Wellcome treatment of potentially life-threatening digitalis
digoxin immune FAB (Rsch. Triangle Park, NC) intoxication in patients who are refractory to
(ovine) management by conventional therapy
Esophageal Varices, Ethamolin~ Reed & Carnrick treatment of bleeding esophageal varices
Bleeding ethanolamine oleate (Piscataway, NJ)
Gallbladder Disease Chenix~t Solvay for patients with radiolucent stones in well
chenodiol (Marietta, GA) opacifying gallbladders, in whom elective surgery
would be undertaken except for the presence of
increased surgical risk due to systemic disease or age
Moctanin Ethitek dissolution of cholesterol gallstones retained in
monooctanoin (Skokie, IL) the common bile duct
Gaucher's Disease Ceredase~ Genzyme Corporation replacement therapy in patients with Gaucher's
alglucerase injection (Cambridge, MA) disease type I
Graft Rejection Leukine~ Immunex treatment of patients who have undergone
sargramostim (Seattle, WA) allogeneic or autologous bone marrow
transplantation and in whom engraftment either
is delayed or fails
Granulomatous Disease, Actimmune~ Genentech treatment of chronic granulomatous disease
Chronic (CGD) interferon gamma-lb (S. San Francisco, CA) (CGD)
Growth Hormone Humatrope~ Eli Lilly long-term treatment of children who have growth
Deficiency somatropin of (Indianapolis, IN) failure due to inadequate secretion of normal
recombinant DNA origin endogenous growth hormone
Protropin~ Genentech long-term treatment of children who have growth
somatrem (S. San Francisco, CA) failure due to a lack of adequate endogenous
growth hormone secretion
Hemophilia AlphaNine~ Alpha Therapeutics control and prevention of bleeding in patients
coagulation factor IX (Los Angeles, CA) with factor IX deficiency due to hemophilia B
(human)
Cyklokapron~ Kabi Pharmacia treatment of patients with hemophilia for shorttranexamic acid (Piscataway, NJ) term use (2 to 8 days) to reduce or prevent
hemorrhage and reduce the need for replacement
therapy during and following tooth extraction
Hyperammonemia Ucephan McGaw, Inc. adjunctive therapy in preventing and treating
benzoate and (Irvine, CA) hyperammonemia in patients with urea cycle
phenylacetate enzymopathy (UCE) due to carbamylphosphate
synthetase, ornithine, transcarbamylase, or
arginosuccinate synthetase deficiency

Hypercalcemia

Didronel~
etidronate disodium

MGI PHARMA
(Minneapolis, MN)

treatment of hypercalcemia of a malignancy
inadequately managed by dietary modification
and/or oral hydration

GaniteTM
gallium nitrate injection

Fujisawa
(Deerfield, IL)

treatment of hypercalcemia of malignancy

t Exclusivity expired as of March 6, 1992.
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