1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
38
Active Trials
200 recruiting
26
Rare Diseases
across 42 areas
0
News (30d)
Quiet
Emory University is a company with 1 orphan drug designation across 26 rare diseases. Active clinical trials in 38 indications. gene therapy candidates in 1 disease. 29 active policy bills affect their portfolio.
Disease-by-disease pipeline progression from orphan designation through clinical trials to FDA approval.
Source: FDA Orphan Drug Designations, ClinicalTrials.gov, Drugs@FDA
| Disease | Drug(s) | Stage |
|---|---|---|
| Alzheimer disease | - | Des.TrialAppr. |
| BCOR ITD sarcoma | l-arginine | Des.TrialAppr. |
| Ewing sarcoma | - | Des.TrialAppr. |
| Ito hypomelanosis | l-arginine | Des.TrialAppr. |
| amyloidosis | - | Des.TrialAppr. |
| brain neoplasm | - | Des.TrialAppr. |
| cancer | - | Des.TrialAppr. |
| cervical cancer | - | Des.TrialAppr. |
| childhood malignant neoplasm | - | Des.TrialAppr. |
| congenital heart disease | - | Des.TrialAppr. |
| cystic fibrosis | - | Des.TrialAppr. |
| diabetes mellitus | - | Des.TrialAppr. |
| epilepsy | - | Des.TrialAppr. |
| hemophilia A | - | Des.TrialAppr. |
| hemophilia B | - | Des.TrialAppr. |
| lupus erythematosus | - | Des.TrialAppr. |
| mitochondrial disease | - | Des.TrialAppr. |
| multiple sclerosis | - | Des.TrialAppr. |
| nevus of Ito | l-arginine | Des.TrialAppr. |
| ocular melanoma | - | Des.TrialAppr. |
| ovarian cancer | - | Des.TrialAppr. |
| pulmonary hypertension | - | Des.TrialAppr. |
| sarcoma | - | Des.TrialAppr. |
| sickle cell disease | l-arginine | Des.TrialAppr. |
| tarsal tunnel syndrome | l-arginine | Des.TrialAppr. |
| thalassemia | - | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
144
overlap in 2+ diseases
1/26
candidate diseases
0
avg importance: 0
29
affecting portfolio
0% of portfolio targets high unmet need diseases
144
overlap in 2+ diseases
1/26
candidate diseases
0
avg importance: 0
29
affecting portfolio