2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
0
Active Trials
200 recruiting
4
Rare Diseases
across 9 areas
0
News (30d)
Quiet
AiroMedica LLC is a company with 2 orphan drug designations across 4 rare diseases. 1 active policy bill affects 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 |
|---|---|---|
| AHDC1-related intellectual disability - obstructive sleep apnea - mild dysmorphism syndrome | recombinant human alpha-1 antitrypsin (rAAT) | Des.TrialAppr. |
| Alstrom syndrome | recombinant human alpha-1 antitrypsin (rAAT) | Des.TrialAppr. |
| cystic fibrosis | recombinant human alpha 1-antitrypsin (rAAT) | Des.TrialAppr. |
| glycogen storage disease II | recombinant human alpha-1 antitrypsin (rAAT) | Des.TrialAppr. |
50% of portfolio targets high unmet need diseases
4
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
1
affecting portfolio
50% of portfolio targets high unmet need diseases
4
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
1
affecting portfolio