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Homogentisate 1,2dioxygenase deficiency — Treatment Landscape & Competitive Intelligence
1 marketed
0 Phase 3
0 Phase 2
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Marketed treatment landscape
| Drug | Generic | Sponsor | Class | Target | Line of therapy | First approval |
|---|---|---|---|---|---|---|
| Orfadin | Orfadin | Cycle Pharmaceuticals Ltd. | 4-hydroxyphenylpyruvate dioxygenase, 4-hydroxyphenylpyruvate dioxygenase |
Phase 3 pipeline
Phase 2 pipeline
Recent regulatory actions (last 90 days)
Upcoming PDUFA dates (next 180 days)
Sponsor landscape
- Cycle Pharmaceuticals Ltd. · 1 drug in Homogentisate 1,2dioxygenase deficiency
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Cite this brief
Drug Landscape (2026). Homogentisate 1,2dioxygenase deficiency — Treatment Landscape & Competitive Intelligence Brief. https://druglandscape.com/ci/disease/homogentisate-1-2dioxygenase-deficiency. Accessed 2026-06-10.
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Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing