2
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
127 recruiting
2
Rare Diseases
across 2 areas
0
News (30d)
Quiet
Percheron Therapeutics Limited is a company with 2 orphan drug designations across 2 rare diseases. 5 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 |
|---|---|---|
| Duchenne muscular dystrophy | 2'-O-(2-methoxyethyl) phosphorothioate antisense oligonucleotide targeting CD49d RNA | Des.TrialAppr. |
| acromegaly | 2'-O-(2-methoxyethyl)phosphorothioate antisense oligonucleotide | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
5
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
5
affecting portfolio