2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
16
Active Trials
200 recruiting
15
Rare Diseases
across 22 areas
0
News (30d)
Quiet
University of Rochester Medical Center is a company with 2 orphan drug designations across 15 rare diseases. Active clinical trials in 16 indications. gene therapy candidates in 1 disease. 28 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. |
| Duchenne muscular dystrophy | deflazacort | Des.TrialAppr. |
| Huntington disease | - | Des.TrialAppr. |
| brain neoplasm | - | Des.TrialAppr. |
| cancer | - | Des.TrialAppr. |
| cervical cancer | - | Des.TrialAppr. |
| childhood malignant neoplasm | - | Des.TrialAppr. |
| epilepsy | - | Des.TrialAppr. |
| lymphoma | - | Des.TrialAppr. |
| multiple system atrophy | - | Des.TrialAppr. |
| muscular dystrophy | - | Des.TrialAppr. |
| nondystrophic myotonia | mexiletine | Des.TrialAppr. |
| pulmonary hypertension | - | Des.TrialAppr. |
| sarcoma | - | Des.TrialAppr. |
| sickle cell disease | - | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
107
overlap in 2+ diseases
1/15
candidate diseases
0
avg importance: 0
28
affecting portfolio
0% of portfolio targets high unmet need diseases
107
overlap in 2+ diseases
1/15
candidate diseases
0
avg importance: 0
28
affecting portfolio