1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
3
Rare Diseases
across 6 areas
0
News (30d)
Quiet
Kira Pharmaceuticals, LLC is a company with 1 orphan drug designation across 3 rare diseases.
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 |
|---|---|---|
| paroxysmal nocturnal hemoglobinuria | Recombinant IgG-fusion protein containing a humanized anti-C5 monoclonal antibody fused with a truncated human complement FH to the heavy chain C-terminus | Des.TrialAppr. |
| paroxysmal nocturnal hemoglobinuria 1 | Recombinant IgG-fusion protein containing a humanized anti-C5 monoclonal antibody fused with a truncated human complement FH to the heavy chain C-terminus | Des.TrialAppr. |
| paroxysmal nocturnal hemoglobinuria 2 | Recombinant IgG-fusion protein containing a humanized anti-C5 monoclonal antibody fused with a truncated human complement FH to the heavy chain C-terminus | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
4
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
4
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio