Orphan Drugs: In Development

DISEASE DRUG NAME COMPANY INDICATION Porphyria Panhematint Abbott amelioration of recurrent attacks of acute interhemin (Abbott Park, IL) mittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and heredita coproporphyria Precocious Puberty Supprelin~ Ortho Pharmaceutical Corp. treatment of central precocious puberty histrelin acetate (Raritan, NJ) Respiratory Distress Exosurf~ NeonataTM Burroughs Wellcome prevention of HMD, also known as respiratory Syndrome (RDS) for Intratracheal (Rsch. Triangle Park, NC) distress syndrome (RDS), in infants born at 32 (Hyaline Membrane Suspension weeks gestation or less Disease-HMD) colfosceril palmitate Exosurf~ NeonatalTM Burroughs Wellcome treatment of established HMD at all gestational for Intratracheal (Rsch. Triangle Park, NC) ages Suspension colfosceril palmitate Survanta~ Ross Laboratories prevention of neonatal RDS beractant (Columbus, OH) Survanta~ Ross Laboratories treatment of neonatal RDS beractant (Columbus, OH) Severe Combined AdagenTM Enzon enzyme replacement therapy for ADA deficiency Immunodeficiency pegademase bovine (S. Plainfield, NJ) in patients with SCID Disease (SCID) Sleeping Sickness Ornidyl~ Marion Merrell Dow treatment of Trypanosoma brucei gambiense eflornithine HCL (Kansas City, MO) infection (sleeping sickness) Strabismus Oculinum~ Allergan treatment of strabismus botulinum toxin type A (Irvine, CA) Tuberculosis Rifadin~ IV Marion Merrell Dow antituberculosis treatment where use of the oral rifampin (Kansas City, MO) form of the drug is not feasible Wilson's Disease Syprine~ Merck treatment of patients with Wilson's disease who trientine HCL (Rahway, NJ) are intolerant or inadequately responsive to penicillamine The content of this report has been obtained through government and industry sources (including FDA and NIH) based on the latest information. The information is current as of March 6, 1992. The information may not be comprehensive. For more specific information about a particular product, contact the individual company directly. For general information, contact the Pharmaceutical Manufacturers Association at (202) 835-3463. (If you did not receive your own copy of this report but would like to, send your request in writing to Editor, "Orphan Drugs in Development, " Pharmaceutical Manufacturers Association, 1100 15th St., NW, Washington, DC 20005.) Copyright ~ 1992 by the Pharmaceutical Manufacturers Association. Permission to reprint is awarded if proper credit is given.

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Title
Orphan Drugs: In Development
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Pharmaceutical Research and Manufacturers of America
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Page 18
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Pharmaceutical Manufacturers Association.
1992
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"Orphan Drugs: In Development." In the digital collection Jon Cohen AIDS Research Collection. https://name.umdl.umich.edu/5571095.0504.010. University of Michigan Library Digital Collections. Accessed June 15, 2025.
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