2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
0
Active Trials
200 recruiting
2
Rare Diseases
across 5 areas
0
News (30d)
Quiet
HemaQuest Pharmaceuticals, Inc. is a company with 2 orphan drug designations across 2 rare diseases. gene therapy candidates in 1 disease. 19 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 |
|---|---|---|
| beta thalassemia | 2 dimethylbutyrate | Des.TrialAppr. |
| sickle cell disease | sodium 2, 2 dimethylbutyrate | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
3
overlap in 2+ diseases
1/2
candidate diseases
0
avg importance: 0
19
affecting portfolio
0% of portfolio targets high unmet need diseases
3
overlap in 2+ diseases
1/2
candidate diseases
0
avg importance: 0
19
affecting portfolio