Sepiapterin
BH4 Deficiency
PTC TherapeuticsSmall moleculePhase 1/2Dormant
CCC
52/ 100
Provisional rating · Methodology v0.2
Rating breakdown
- Clinical Evidence
- 37/ 100
- Competitive Position
- 95/ 100
Competitive context
0 Active assets in the BH4 Deficiency cohort.
| Asset / Sponsor | Phase | Rating |
|---|---|---|
(6S)-L-ERYTHRO-TETRAHYDROBIOPTERIN University of Massachusetts, Amherst | Phase 1 | CC |
Sapropterin Dihydrochloride BioMarin Pharmaceutical | NR | |
Phenoptin BioMarin Pharmaceutical | Phase 2 | NR |
Same molecule, other indications
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