Orphan Drugs: In Development

"able o

iten

Cover Article: 189 Medicines in Testing for Rare Diseases.. Page 1
Some Key Areas of Orphan Drug Research............ Page 2
Orphan Drugs in Development..................... Page 3
Approved Orphan Drugs......................... Page 15

G lossary..................................... Page 19
PMA Commission Seeks Corporate Parents for
Orphan Drugs in Development.................. Page 23
List of Organizations Presenting the Report with PMA... Page 24

m4o

Le?

'eas of Orphan Drug Research

Orphan drug research is a highly complex area of medicine. This survey report alone shows that orphan drugs are in development for 242
rare conditions in 122 disease categories. To give some focus to this complexity, we have identified six of the important areas of orphan drug
research. They are listed below as: AIDS and AIDS-related conditions, cancers, childhood diseases, genetic disorders, neuromuscular disorders,
and diseases of women.
Under each of the six areas are disease categories that appear in the first column of the chart that begins on page 3. For particulars about
the drugs in each category, please consult the "Disease" column in the chart.

AIDS, AIDS-RELATED CONDITIONS
(20 drugs in development)
Acquired Immune Deficiency Syndrome
(AIDS)
Anorexia
Cachexia
Cytomegalovirus
Diarrhea
Mycobacterium Avium Disease
Neutropenia
Pneumocystis Carinii Pneumonia (PCP)
CANCERS (58 drugs in development)
Cancer, Adrenal
Cancer, Bladder
Cancer, Bone
Cancer, Brain
Cancer, Breast
Cancer, Cervical
Cancer, Colorectal
Cancer, Esophageal
Cancer, Germ Cell
Cancer, Head and Neck
Cancer, Hepatic
Cancer, Leukemia
Cancer, Lung
Cancer, Lymphoma
Cancer, Melanoma
Cancer, Multiple Myeloma
Cancer, Ovarian
Cancer, Pancreatic
Cancer, Renal
Cancer, Soft Tissue
Cancer, Thyroid
Chemotherapy Complications
Graft Rejection
Graft Vs. Host Disease
Neutropenia
Pain
Xerostomia

CHILDHOOD DISEASES
(50 drugs in development)
Anemia
Botulism, Infant
Cancer, Leukemia
Carnitine Deficiency
Cerebral Palsy
Charcot-Marie-Tooth Disease
Cystic Fibrosis
Cystinosis
Dysplasia, Bronchopulmonary
Epilepsy
Fabry's Disease
Gaucher's Disease
Growth Hormone Deficiency
Hemophilia
Hyperbilirubinemia
Lactic Acidosis, Congenital
Lennox-Gastaut Syndrome
Low Birth Weight
Muscular Dystrophy
Myelodysplastic Syndromes
Precocious Puberty
Puberty, Delayed
Respiratory Distress Syndrome, Neonatal
(RDS)
Respiratory Syncytial Virus (RSV)
Short Stature
Sickle Cell Anemia
Spasticity, Intractable
Turner's Syndrome
Von Willebrand's Disease
DISEASES OF WOMEN
(17 drugs in development)
Amenorrhea
Cancer, Breast
Cancer, Cervical
Cancer, Ovarian
Cystitis, Interstitial

Infertility
Sjogren's Syndrome
Turner's Syndrome
Vulvar Dystrophies
GENETIC DISORDERS
(35 drugs in development)
Alpha-1 Antitrypsin Deficiency
Amyotrophic Lateral Sclerosis
Anemia
Carnitine Deficiency
Charcot-Marie-Tooth Disease
Cystic Fibrosis
Cystinosis
Fabry's Disease
Gaucher's Disease
Growth Hormone Deficiency
Hemophilia
Hypercholesterolemia
Lactic Acidosis, Congenital
Multiple Sclerosis
Muscular Dystrophy
Porphyria
Sickle Cell Anemia
Turner's Syndrome
Von Willebrand's Disease
Wilson's Disease
NEUROMUSCULAR DISORDERS
(15 drugs in development)
Amyotrophic Lateral Sclerosis
Causalgia
Cerebral Palsy
Lambert-Eaton Syndrome
Lennox-Gastaut Syndrome
Multiple Sclerosis
Muscular Dystrophy
Myoclonus
Spasticity, Intractable