Sephience

(Sepiapterin)

Phase 3Small molecule

PPTC Therapeutics

Sephience is one of 7 small molecule programs targeting Phenylketonuria. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

PhenylketonuriaRecruiting

PhenylketonuriaPhase 3

Recruiting

Competition

Competitive Landscape

8 competitors in Phenylketonuria

#DrugCompanyPhaseTrials

Avelox

Bayer

Approved

—2

L-Carnitine

IIperboreal Pharma Srl

Phase 3

1 3

CEB-01

BioMarin Pharmaceutical

Phase 3

1 4

Sephience

PPTC Therapeutics

Phase 3

2 5

Pegvaliase

BioMarin Pharmaceutical

Preclinical

—6

AG-181

AAgios Pharmaceuticals, Inc.

Phase 1

1 7

Repinatrabit

Otsuka

Phase 1

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Recruiting

Phenylketonuria

NCT07728032

Q1 2031

Phase 3Recruiting

Phenylketonuria

NCT06302348

Clinical

Trials by Indication[1]

Phenylketonuria2 trials

A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin

Recruiting·PPTC Therapeutics·Phase 3·NCT06302348·56 enrolled

Impact Of A Phenylalanine-Restricted Diet On The Microbiota Composition And Metabolome Of Children With PKU

Recruiting·BBirmingham Women's and Children's NHS Foundation Trust··NCT07728032·148 enrolled

Regulatory

FDA Approval Timeline[4]

Jul 28, 2025

SEPHIENCE

NDA NDA219666

Sources

References

[1]
ClinicalTrials.gov — 2 trial records for Sepiapterin. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Sepiapterin compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Sepiapterin. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA219666). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Sephience. U.S. Food & Drug Administration.
[6]
SEC EDGAR — PTC Therapeutics company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: PPTC Therapeutics

Patent Landscape[5]

Patent Cliff

Apr 16, 2038

Earliest patent expiration — generic entry possible after this date

Patent #Expires

11072614Apr 16, 2038

12213982Sep 4, 2038

11752154Sep 4, 2038

12257252Mar 6, 2042

Sephience

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2026

Q1 2031

Trials by Indication[1]

Phenylketonuria2 trials

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

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