4
Orphan Designations
0 approved, 4 designated
0
FDA Approvals
0
Active Trials
21 recruiting
1
Rare Diseases
across 3 areas
0
News (30d)
Quiet
JCR Pharmaceuticals Co., Ltd. is a company with 4 orphan drug designations across 1 rare disease.
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 |
|---|---|---|
| mucopolysaccharidosis type 1 | Recombinant fusion protein of human alpha-L-iduronidase (IDUA) and Fab fragment of a humanized monoclonal antibody targeting human transferrin receptor (hTfR). | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/1
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/1
candidate diseases
0
avg importance: 0
0
affecting portfolio