1
Orphan Designations
0 approved
0
FDA Approvals
0
Active Trials
3
Rare Diseases
across 6 areas
0
News (30d)
Quiet
Laboratoire francais du Fractionnement et des Biot is a company with 1 orphan drug designation across 3 rare diseases. gene therapy candidates in 1 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 |
|---|---|---|
| ALG2-congenital disorder of glycosylation | human coagulation factor XI | Des.TrialAppr. |
| MGAT2-congenital disorder of glycosylation | human coagulation factor XI | Des.TrialAppr. |
| isolated growth hormone deficiency type IA | human coagulation factor XI | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
1/3
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
1/3
candidate diseases
0
avg importance: 0
0
affecting portfolio