Drug Profile
Sepiapterin - PTC Therapeutics
Alternative Names: CNSA 001; PTC-923Latest Information Update: 12 Mar 2024
Price :
$50
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At a glance
- Originator Censa Pharmaceuticals
- Developer Censa Pharmaceuticals; PTC Therapeutics
- Class Amines; Gastrokinetics; Ketones; Pteridines; Small molecules; Vasodilators
- Mechanism of Action Mixed function oxygenase modulators
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Phase III Phenylketonuria
- Phase II Diabetic gastroparesis
- No development reported Dystonia
Most Recent Events
- 29 Feb 2024 PTC Therapeutics plans the phase IIIb EPIPHENY trial for Phenylketonuria (In Children) in the US (PO, Suspension) (NCT06302348)
- 29 Feb 2024 PTC Therapeutics intention to submit Marketing Authorization Application (MAA) to European Medicines Agency (EMA) for Phenylketonuria in March 2024
- 08 Jan 2024 PTC Therapeutics intention to submit NDA to US FDA for Phenylketonuria in no later than the third quarter 2024