1
Orphan Designations
0 approved
0
FDA Approvals
4
Active Trials
200 recruiting
5
Rare Diseases
across 10 areas
0
News (30d)
Quiet
Genzyme, a Sanofi Company is a company with 1 orphan drug designation across 5 rare diseases. Active clinical trials in 4 indications.
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 |
|---|---|---|
| brain neoplasm | - | Des.TrialAppr. |
| hemophilia A | - | Des.TrialAppr. |
| hemophilia B | - | Des.TrialAppr. |
| mucopolysaccharidosis type 1 | - | Des.TrialAppr. |
| primary focal segmental glomerulosclerosis | fresolimumab | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
24
overlap in 2+ diseases
0/5
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
24
overlap in 2+ diseases
0/5
candidate diseases
0
avg importance: 0
0
affecting portfolio