2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
0
Active Trials
2
Rare Diseases
across 3 areas
0
News (30d)
Quiet
Laboratoires Théa is a company with 2 orphan drug designations across 2 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 |
|---|---|---|
| Leber congenital amaurosis | antisense oligonucleotide complementary to the exonic splicer enhancer sequence at intron 26 of the centrosomal protein 290 pre-mRNA | Des.TrialAppr. |
| retinitis pigmentosa | 2¿-O-(2-methoxyethyl) modified antisense oligonucleotide targeting exon 13 in the USH2A gene | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio