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 [phone].
(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.