1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
1
Rare Diseases
across 1 areas
0
News (30d)
Quiet
Sanofi Genzyme, A Sanofi Company is a company with 1 orphan drug designation 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 |
|---|---|---|
| osteogenesis imperfecta | anti-human transforming growth factor beta (TGF-Beta) monoclonal antibody (mAb), based on the amino acid sequence of the human monoclonal antibody fresolimumab (GC1008) with the exception of one serine to proline substitution | 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