Orphan drugs share in common only one major characteristic:They are intended to treat conditions or diseases with patientpopulations no greater than 200,000. Some drugs are grantedthat status while still in clinical trials. Others don't get the noduntil they are approved for marketing. Sometimes it's a racebetween competing products. Some are monoclonal antibodies,while others are hormones, enzymes or amino acids.Listed below is a sample -- not intended to be comprehensive-- of the compounds that FDA designated as orphan drugs in1992.
Atrial natriuretic peptideFor kidney transplantation and for treating acute kidneyfailure.Scios Nova Inc. (NASDAQ:SCIO), Mountain View, Calif.
TYPE B BOTULINUM TOXIN (AN-072)
For treating cervical dystonia (involuntary muscle contraction)Athena Neurosciences Inc. (NASDAQ:ATHN), South SanFrancisco, Calif.
CFTR (cystic fibrosis transmembrane regulator) Protein
For replacing the gene product damaged in cystic fibrosis.Genzyme Corp. (NASDAQ:GENZ), Cambridge, Mass.
CNTF (ciliary neurotrophic factor)
For treating motor neuron disease: amyotrophic lateral sclerosis(ALS, or Lou Gehrig's disease), progressive muscular atrophy,progressive bulbar palsy, primary lateral sclerosis and spinalmuscular atrophies.Regeneron Pharmaceuticals Inc. (NASDAQ:REGN), Tarrytown,N.Y.; also Syntex-Synergen Inc. Neuroscience Joint Venture(NASDAQ:SYGN), Boulder, Colo.The first product to get marketing approval receives sevenyears marketing exclusivity
GENE THERAPY FOR CF
Insert CFTR gene into patient's cellsFirst orphan drug designation for a gene therapy.Genzyme Corp.
Technitium-99m labeled monoclonal antibodyImaging agent for non-Hodgkin's B-cell lymphoma, acute B-celllymphoblastic leukemia and chronic B-cell lymphocyticleukemia.Immunomedics Inc. (NASDAQ:IMMU), Warren, N.J.
A form of pilocarpine hydrochlorideFor treating xerostomia (dry mouth) and keratoconjunctivitissicca (dry eye) in Sjogren's syndrome patients.MGI Pharma (NASDAQ:MOGN), Minneapolis
Antibody panel for treating non-Hodgkin's B-cell lymphomasand leukemias.Idec Pharmaceuticals Corp. (NASDAQ:IDPH), La Jolla, Calif.Orphan drug status begins with actual marketing date
Amino acidFor treating neurological disease: familial spastic paraparesis.Interneuron Pharmaceuticals Inc. (NASDAQ:IPIC), Lexington,Mass.
Recombinant human thyroid stimulating hormoneAs adjunct in diagnosing thyroid cancer.Genzyme (NASDAQ;GENZ) and Neozyme (NASDAQ:NEOZ), both ofCambridge, Mass.
Enzymatic debridement agent for treating severe burns.Genzyme (NASDAQ:GENZ) and Neozyme (NASDAQ:NEOZ), both ofCambridge, Mass.
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