Orphan Drugs: In Development

DISEASE DRUG NAME COMPANY INDICATION
Hyperphosphatemia Phos-Lo~ Braintree Laboratories treatment of hyperphosphatemia in end-stage
calcium acetate (Braintree, MA) renal failure
Hypocalcemia Parathar~ Rhone-Poulenc Rorer diagnostic agent to assist in establishing the
teriparatide (Collegeville, PA) diagnosis in patients presenting with clinical and
laboratory evidence of hypocalcemia due to either
hypoparathyroidism or pseudohypoparathyroidism
Hypocitraturia Urocit-K~ Mission Pharmacal control and prevention of calcium renal stones in
potassium citrate (San Antonio, TX) patients with hypocitraturia
Keratoconjunctivitis, Opticrom~t Fisons Corporation treatment of vernal keratoconjunctivitis
Vernal cromolyn sodium 4% (Rochester, NY)
ophthalmic solution
Lead Poisoning ChemetTM McNeil Consumer Products treatment of lead poisoning in children
succimer (Spring House, PA)
Leprosy Lamprene~ CIBA-GEIGY treatment of lepromatous leprosy, including that
clofazimine (Summit, NJ) which is dapsone-resistant and that which is
complicated by erythema nodosum leprosum
Leukapheresis Pentaspan~ Du Pont Merck adjunct in leukapheresis to improve the harvesting
pentastarch (Wilmington, DE) and increase the yield of leukocytes by centrifugal
means
Malaria Lariam~ Hoffmann-La Roche prophylaxis of Plasmodium falciparum malaria
mefloquine HCL (Nutley, NJ) that is resistant to other available drugs
Lariam~ Hoffmann-La Roche treatment of acute malaria due to Plasmodium
mefloquine HCL (Nutley, NJ) falciparum and Plasmodium vivax
Mastocytosis Gastrocom~ Fisons Corporation treatment of mastocytosis
cromolyn sodium (Rochester, NY)
Nephrolithiasis Renacidin~ Irrigation United-Guardian treatment of renal and bladder calculi of the
citric acid, glucono- (Smithtown, NY) apatite or struvite variety
delta-lactone,
magnesium carbonate
Thiola~ Mission Pharmacal control and prevention of cystine nephrolithiasis
tiopronin (San Antonio, TX) in patients with homozygous cystinuria
Urocit-K~ Mission Pharmacal control and prevention of uric acid nephrolithiasis
potassium citrate (San Antonio, TX)
Urocit-K~ Mission Pharmacal avoidance of the complication of calcium stone
potassium citrate (San Antonio, TX) formation in patients with uric lithiasis
Neutropenia Leukine~ Immunex treatment of neutropenia associated with bone
sargramostim (Seattle, WA) marrow transplantation in patients with nonHodgkin's lymphoma, Hodgkin's disease and ALL
Opioid Dependency TrexanMt Du Pont Merck blockade of the pharmacological effects of
naltrexone HCL (Wilmington, DE) exogenously administered opioids as an adjunct
to the maintenance of the opioid-free state in
detoxified formerly opioid-dependent individuals
Paget's Disease Cibacalcin~ CIBA-GEIGY treatment of symptomatic Paget's disease of bone
calcitonin-human (Summit, NJ) (osteitis deformans)
Pain InfumorphTM Elkins-Sinn for use in microinfusion devices for intraspinal
preservative-free (Cherry Hill, NJ) administration in the treatment of intractable
morphine sulfate chronic pain
solution
Parkinson's Disease Eldepryl~ Somerset adjuvant to levodopa and carbidopa treatment of
selegiline HCL (Denville, NJ) idiopathic Parkinson's disease (paralysis agitans),
post-encephalitic parkinsonism, and symptomatic
parkinsonism
Pneumocystis Carinii NebuPent~ Fujisawa prevention of PCP in patients at high risk of
Pneumonia (PCP) pentamidine isethionate (Deerfield, IL) developing this disease
Pentam~ 300t Fujisawa treatment of PCP

pentamidine isethionate (Deerfield, IL)
Polycystic Ovarian Metrodin~ Serono Laboratories induction of ovulation in patients with polycystic
Disease urofollitropin (Norwell, MA) ovarian disease who have an elevated LH/FSH
for injection ratio and who have failed to respond to adequate
clomiphene citrate therapy
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