Orphan Drugs: In Development

DEVELOPMENT
STATUS

DISEASE

DRUG NAME

SPONSOR

FDA OFFICIAL DESIGNATION

Hyperphosphatemia

calcium acetate

Pharmedic
(Wheeling, IL)

treatment of hyperphosphatemia in
patients with end-stage renal disease
(ESRD)

Phase I

calcium carbonate
(chewable and tablet)

R&D Laboratories
(Marina del Rey, CA)

treatment of hyperphosphatemia in
patients with ESRD

Phase III

Hypertension, Flolan~ Burroughs Wellcome treatment of primary pulmonary Phase III
Pulmonary epoprostenol (Rsch. Triangle Park, NC) hypertension (PPH)
Hypotension, Amatine~ Roberts Pharmaceutical treatment of idiopathic orthostatic application submitted
Orthostatic midodrine HCL (Eatontown, NJ) hypotension
Infertility Geref~ Serono Laboratories adjunct to gonadotropin therapy in Phase III
sermorelin acetate (Norwell, MA) the induction of ovulation in women
with anovulatory or oligovulatory
infertility who fail to ovulate in
response to adequate treatment with
clomiphene citrate alone and
gonadotropin therapy alone
Lactic Acidosis, DCA Peter Stacpoole, M.D. treatment of congenital lactic Phase III
Congenital sodium dichloro- Univ. of Florida acidosis
acetate (Gainesville, FL)
Lambert-Eaton 3,4-diaminopyridine Jacobus Pharmaceutical treatment of Lambert-Eaton Phase I
Syndrome (Princeton, NJ) myasthenic syndrome
dynamine Mayo Clinic treatment of Lambert-Eaton Phase II
(Rochester, MN) myasthenic syndrome
Lennox-Gastaut felbamate Wallace treatment of Lennox-Gastaut application submitted
Syndrome (Cranbury, NJ) syndrome
Low Birth Weight multivitamin Astra Pharmaceuticals for establishment and maintenance Phase I
neonatal formula (Westborough, MA) of total parenteral nutrition in very
low birth weight infants
Malaria Halfan SmithKline Beecham treatment of mild to moderate acute in clinical trials
halofantrine (Philadelphia, PA) malaria caused by susceptible strains
of p. falciparum and p. vivax
Mephaquin Mepha AG prevention of chloroquine-resistant Phase III
mefloquine HCL (Dornach, Switzerland) falciparum malaria
Mercury Chemet~ McNeil Consumer treatment of mercury intoxication Phase III
Intoxication succimer Products
(Ft. Washington, PA)
Multiple Sclerosis 4-aminopyridine Elan Pharmaceutical relief of symptoms of multiple Phase II
(Gainesville, GA) sclerosis
Betaseron~ Berlex treatment of multiple sclerosis Phase III
interferon beta, (Wayne, NJ)
recombinant human
Centara Centocor treatment of multiple sclerosis Phase II
anti-CD4 (Malvern, PA)
copolymer 1 (COP 1) TAG Pharmaceuticals/ treatment of multiple sclerosis Phase III
Lemmon Company
(Sellersville, PA)
Muscular Sanorex Platon J. Collip, M.D. treatment of Duchenne muscular application submitted
Dystrophy mazindol (Jesup, GA) dystrophy (DMD)
Mycobacterium gentamicin liposome The Liposome Company treatment of disseminated Phase II
Avium Disease injection (Princeton, NJ) Mycobacterium avium(TLC G-65) intracellulare infection
Mycobutin Adria prevention of Mycobacterium application submitted
rifabuten (Columbus, OH) avium complex (MAC) disease in
patients with AIDS and in patients
with CD4 counts less than 200/MM3
Mycobutin Adria treatment of disseminated Phase III
rifabuten (Columbus, OH) Mycobacterium avium complex
(MAC) disease
Myelodysplastic Leucomax~ Schering-Plough treatment of myelodysplastic Phase III
Syndromes granulocyte/ (Madison, NJ) syndromes
macrophage-colony
stimulating factor
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