Table 3 Top 25 diseases with the most designations with at least one orphan drug approval, 1983–2022
Disease | Therapeutic area | Initial orphan drug approvals | Designations |
|---|---|---|---|
HIV infectious disease* | Infectious Diseases | 23 | 92 |
Multiple myeloma | Oncology | 19 | 130 |
Non-small cell lung carcinoma | Oncology | 18 | 35 |
Metastatic melanoma | Oncology | 16 | 120 |
Acute myeloid leukemia | Oncology | 14 | 183 |
B-cell chronic lymphocytic leukemia | Oncology | 12 | 57 |
Hepatocellular carcinoma | Oncology | 11 | 89 |
Follicular lymphoma | Oncology | 11 | 37 |
Isolated congenital growth hormone deficiency | Endocrinology | 10 | 23 |
Pulmonary arterial hypertension | Vascular | 9 | 58 |
Hemophilia B | Hematology | 9 | 23 |
Ovarian cancer* | Oncology | 8 | 91 |
Diffuse large B-cell lymphoma | Oncology | 8 | 33 |
Cystic fibrosis | Pulmonology | 8 | 108 |
Chronic myelogenous leukemia | Oncology | 8 | 38 |
Acute lymphoblastic leukemia | Oncology | 8 | 55 |
Solid organ transplant rejection | Transplant | 7 | 49 |
Thyroid cancer | Oncology | 7 | 13 |
Lennox–Gastaut syndrome | Neurology | 7 | 16 |
Soft tissue sarcoma | Oncology | 6 | 56 |
Neuroendocrine neoplasm | Oncology | 6 | 24 |
Malaria | Infectious Diseases | 6 | 28 |
Mantle cell lymphoma | Oncology | 6 | 23 |
Homozygous familial hypercholesterolemia | Metabolism | 6 | 16 |
Immune thrombocytopenic purpura | Hematology | 6 | 22 |