2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
1
Active Trials
200 recruiting
3
Rare Diseases
across 7 areas
0
News (30d)
Quiet
Disc Medicine is a company with 2 orphan drug designations across 3 rare diseases. Active clinical trials in 1 indication. 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 |
|---|---|---|
| acquired polycythemia vera | recombinant humanized monoclonal antibody targeting transmembrane serine protease matripase-6 | Des.TrialAppr. |
| erythropoietic protoporphyria | bitopertin | Des.TrialAppr. |
| sickle cell disease | - | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
1/3
candidate diseases
0
avg importance: 0
19
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
1/3
candidate diseases
0
avg importance: 0
19
affecting portfolio