Orphan Drugs: In Development

DISEASE
Poisoning,
Chemical

DRUG NAME
4-MP
4-methylpyrazole

SPONSOR
Louisiana State University
Medical Center
(Shreveport, LA)

FDA OFFICIAL DESIGNATION
treatment of methanol, ethylene
glycol, 2-butoxyethanol or
2-methoxyethanol poisoning

DEVELOPMENT
STATUS
Phase I/II

Polycythemia Agrelin~ Roberts Pharmaceutical treatment of polycythemia vera Phase III
Vera anagrelide (Eatontown, NJ)
Porphyria histrelin Karl Anderson, M.D. treatment of acute intermittent Phase III
University of Texas porphyria, hereditary
Medical Branch coproporphyria, and variegata
(Galveston, TX) porphyria
Precocious Lupron~ TAP Pharmaceuticals treatment of central precocious application submitted
Puberty leuprolide acetate (Deerfield, IL) puberty
Somagard~ Roberts Pharmaceutical treatment of central precocious Phase III
deslorelin (Eatontown, NJ) puberty
Synarel~ Syntex treatment of central precocious application submitted
nafarelin acetate (Palo Alto, CA) puberty
Puberty, Delayed Androtest-SLTM Gynex Pharmaceuticals treatment of constitutional delay of Phase II
sublingual (Vernon Hills, IL) growth and puberty in boys
testosterone
OxandrinTM Gynex Pharmaceuticals treatment of constitutional delay of Phase III
oxandrolone (Vernon Hills, IL) growth and puberty
Raynaud's iloprost Berlex treatment of Raynaud's phenomenon application submitted
Disease (Wayne, NJ) secondary to systemic sclerosis
Respiratory ALECTM Britannia Pharmaceuticals prevention and treatment of neonatal Phase III
Distress (Surrey, United Kingdom) respiratory distress syndrome (RDS)
Syndrome Infasurf~ Ony prevention and treatment of Phase III
(RDS), calf lung surfactant (Buffalo, NY) respiratory failure due to pulmonary
Neonatal extract (CLSE) surfactant deficiency in preterm infants
Respiratory Hypermune RSVTM MedImmune prophylaxis of respiratory syncytial Phase III
Syncytial Virus respiratory syncytial (Gaithersburg, MD) virus (RSV), lower respiratory tract
Disease virus immune infections in infants and young
globulin (human) children at high risk of RSV disease
Hypermune RSVTM MedImmune treatment of respiratory syncytial Phase III
respiratory syncytial (Gaithersburg, MD) virus (RSV), lower respiratory tract
virus immune infections in hospitalized infants
globulin (human) and young children
Retinitis Cronassial~ Fidia Pharmaceutical treatment of retinitis pigmentosa Phase II
Pigmentosa gangliosides as (Washington, DC)
sodium salts
Seizures Diastat Upsher-Smith treatment of acute repetitive Phase II/III
diazepam viscous (Minneapolis, MN) seizures
solution
Sexual papaverine Pharmedic treatment of sexual dysfunction in Phase II
Dysfunction topical gel (Wheeling, IL) spinal cord injury patients
Short Stature Nutropin Genentech treatment of short stature associated Phase III
somatropin for (S. San Francisco, CA) with chronic renal insufficiency
injection
Sickle Cell BW 12C Burroughs Wellcome treatment of sickle cell disease Phase II
Anemia (Rsch. Triangle Park, NC)
RheothRx~ Burroughs Wellcome treatment of sickle cell crisis Phase II
poloxamer 188 (Rsch. Triangle Park, NC)
Sjogren's cyclosporine University of Georgia treatment of severe kerato- Phase II
Syndrome ophthalmic (Athens, GA) conjunctivitis sicca associated with
Sjogren's syndrome
Soasticitv. baclofen Infusaid. Inc. treatment of intractable spasticitv Phase III

Intractable

(Norwood, MA)

L.... a a................ r I.. a..
due to multiple sclerosis or spinal
cord injury

Lioresal~ Medtronic, Inc. treatment of intractable spasticity application submitted
Intrathecal (Minneapolis, MN) caused by spinal cord injury or
baclofen multiple sclerosis
Neuralgon Mericon Industries treatment of intractable spasticity in Phase II

v
L-baclofen (Peoria, IL)

A
children with cerebral palsy