2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
0
Active Trials
4
Rare Diseases
across 5 areas
0
News (30d)
Quiet
COUR Pharmaceuticals Development Company, Inc. is a company with 2 orphan drug designations across 4 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 |
|---|---|---|
| juvenile myasthenia gravis | composed of seven antigenic peptides derived from the acetylcholine receptor alpha AChR (AChRalpha) and epsilon (AChRepsilon) subunits dispersed within a negatively charged (-30 to -60 mV) polymer matrix of poly(D,L-lactide-co-glycolide) (PGLA, 50:50 acid-end group) particles (400 to 800 nm mean size) at a target concentration of approximately 1 microgram of each AChR peptide comprising AChR peptide pool drug substance per mg of PLGA | Des.TrialAppr. |
| myasthenia gravis | composed of seven antigenic peptides derived from the acetylcholine receptor alpha AChR (AChRalpha) and epsilon (AChRepsilon) subunits dispersed within a negatively charged (-30 to -60 mV) polymer matrix of poly(D,L-lactide-co-glycolide) (PGLA, 50:50 acid-end group) particles (400 to 800 nm mean size) at a target concentration of approximately 1 microgram of each AChR peptide comprising AChR peptide pool drug substance per mg of PLGA | Des.TrialAppr. |
| primary biliary cholangitis | PDC-E2-peptide drug substance | Des.TrialAppr. |
| transient neonatal myasthenia gravis | composed of seven antigenic peptides derived from the acetylcholine receptor alpha AChR (AChRalpha) and epsilon (AChRepsilon) subunits dispersed within a negatively charged (-30 to -60 mV) polymer matrix of poly(D,L-lactide-co-glycolide) (PGLA, 50:50 acid-end group) particles (400 to 800 nm mean size) at a target concentration of approximately 1 microgram of each AChR peptide comprising AChR peptide pool drug substance per mg of PLGA | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
2
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
2
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
0
affecting portfolio