Orphan Drugs: In Development

DISEASE
Myelodysplastic
Syndromes
(continued)

DRUG NAME
Neupogen~
filigrastim

SPONSOR FDA OFFICIAL DESIGNATION
Amgen treatment of myelodysplastic
(Thousand Oaks, CA) syndrome

DEVELOPMENT
STATUS
Phase III

Myoclonus L-5 hydroxytrypto- Bolar Pharmaceuticals treatment of postanoxic intention Phase II
phan (L-5HTP) (Copiague, NY) myoclonus
Nerve Repair leupeptin Lawrence Hurst, M.D. adjunct to microsurgical peripheral Phase I
(Stony Brook, NY) nerve repair
Neutropenia Leucomax~ Schering-Plough treatment of neutropenia associated Phase III
granulocyte/ (Madison, NJ) with bone marrow transplants
macrophage-colony
stimulating factor
Leucomax~ Schering-Plough treatment of AIDS patients with Phase II/III
granulocyte/ (Madison, NJ) neutropenia due to either the disease
macrophage-colony itself or zidovudine (azidothymidinestimulating factor AZT) or ganciclovir (DHPG) therapy
Neupogen~ Amgen treatment of neutropenia associated Phase III
filigrastim (Thousand Oaks, CA) with bone marrow transplants
Neupogen~ Amgen treatment of patients with severe Phase III
filigrastim (Thousand Oaks, CA) chronic neutropenia (absolute
neutrophil count less than 500/mm)
Osteomyelitis Septopal~ EM Industries treatment of chronic osteomyelitis Phase III
gentamicin (Hawthorne, NY) of post-traumatic, post-operative or
impregnated PMMA hematogenous origin
beads on surgical wire
Paget's Disease Miacalcin Nasal Sandoz treatment of symptomatic Paget's Phase III
Spray (East Hanover, NJ) disease of bone (osteitis deformans)
calcitonin salmon
nasal spray
Pain clonidine HCL Fujisawa epidural administration for the Phase III
(epidural injection) (Deerfield, IL) treatment of pain in cancer patients
tolerant to, or unresponsive to,
intraspinal opiates
Papillomavirus/ Wellferon~ Burroughs Wellcome treatment of human papillomavirus application submitted
Papillomatosis interferon alfa-nl (Rsch. Triangle Park, NC) (HPV) in patients with severe
resistant/recurrent respiratory
(laryngeal) papillomatosis
Penicillin Pre-Pen/MDM Schwarz Pharma assessing the risk of administering Phase III
Sensitivity benzylpenicillin, Kremers Urban penicillin when it is the preferred
benzylpenicilloic, (Milwaukee, WI) drug of choice in adult patients
benzylpenilloic acid who have previously received
penicillin and have a history of
clinical sensitivity
Pneumocystis atovaquone Burroughs Wellcome prevention of PCP in high-risk, Phase III

Carinii
Pneumonia
(PCP)

(566C80)

(Rsch. Triangle Park, NC)v
(Rsch. Triangle Park, NC)

HIV-infected patients defined by a
history of one or more episodes of
PCP and/or a peripheral CD4 +
(T4 helper/inducer) lymphocyte
count less than or equal to 200/mm3

atovaquone Burroughs Wellcome treatment of AIDS-related PCP Phase III
(566C80) (Rsch. Triangle Park, NC)
Dapsone Jacobus Pharmaceutical prophylaxis for PCP Phase III
(Princeton, NJ)
Dapsone Jacobus Pharmaceutical treatment of PCP in combination Phase III
(Princeton, NJ) with trimethoprim
pentamidine Rhone-Poulenc Rorer treatment of PCP application submitted
isethionate (Collegeville, PA)
PneumopentM Fisons Corporation prevention of PCP in patients at application submitted
pentamidine (Bedford, MA) high risk of developing this disease
isethionate
(inhalation)
trimetrexate U.S. Bioscience treatment of PCP in AIDS patients Phase III

glucuronate

(West Conshohocken, PA)

12