0
Orphan Designations
1
FDA Approvals
Latest: Luxturna (2017)
0
Active Trials
1
Rare Diseases
across 3 areas
0
News (30d)
Quiet
Spark Therapeutics is a company with 0 orphan drug designations across 1 rare disease, including 1 FDA-approved therapy.
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 |
|---|---|---|
| inherited retinal dystrophy | Luxturna | 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