List of FDA Orphan Drugs
GARD has information from the Food and Drug Administration (FDA) on treatments approved for rare diseases, known as orphan products/drugs. The Orphan Drug Act was passed in 1983 to give drug companies incentives to develop treatments for rare diseases. The FDA Office of Orphan Products Development determines if a drug qualifies as an orphan product. The following information comes from the FDA database of orphan drug designations and approvals. You can output the data into an Excel spreadsheet from the FDA website. More information on orphan drug development is available from Global Genes and Orphanet.
P
- Paget disease of bone
- Paget disease of bone, familial
- Pancreatic cancer
- Panuveitis
- Papillary renal cell carcinoma
- Papillary thyroid carcinoma
- Paraganglioma and gastric stromal sarcoma
- Parathyroid carcinoma
- Parkinson disease
- Parkinson disease type 3
- Parkinson disease type 9
- Paroxysmal nocturnal hemoglobinuria
- Pars planitis
- Patent ductus arteriosus
- Pediatric Crohn's disease
- Pediatric hypertension
- Pediatric ulcerative colitis
- Pemphigus vulgaris
- Peripheral T-cell lymphoma
- Peroxisome biogenesis disorder-Zellweger syndrome spectrum
- Phenylketonuria
- Pheochromocytoma
- Pheochromocytoma, childhood
- Pheochromocytoma-islet cell tumor syndrome
- Philadelphia-negative chronic myeloid leukemia
- Pneumocystic carinii pneumonia
- Polyarticular onset juvenile idiopathic arthritis
- Polycythemia vera
- Posterior uveitis
- Prader-Willi syndrome
- Precocious puberty
- Premature aging Okamoto type
- Primary biliary cholangitis
- Primary carnitine deficiency
- Primary hyperparathyroidism
- Primary liver cancer
- Primary malignant melanoma of the cervix
- Primary malignant melanoma of the conjunctiva
- Protein C deficiency
- Psoriatic juvenile idiopathic arthritis
- Pulmonary arterial hypertension
- Pure autonomic failure
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