Sephience

(Sepiapterin)

Phase 3Small molecule

PPTC Therapeutics

Sephience is one of 17 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

PhenylketonuriaActive, not recruiting

PhenylketonuriaPhase 3

Active, not recruiting

Competition

Competitive Landscape

29 competitors in Phenylketonuria

#DrugCompanyPhaseTrials

Avelox

Bayer

Approved

—2

L-Carnitine

IIperboreal Pharma Srl

Approved

1 3

BMN 333

BioMarin Pharmaceutical

Phase 2

1 4

CEB-01

BioMarin Pharmaceutical

Phase 3

1 5

Sephience

PPTC Therapeutics

Phase 3

1 6

PTC923

PPTC Therapeutics

Phase 3

2 7

Kuvan

BioMarin Pharmaceutical

Preclinical

5 8

Immunosuppression

NNational Heart, Lung, and Blood Institute (NHLBI)

Phase 2

1 9

Kuvan

BioMarin Pharmaceutical

Preclinical

5 10

Tetrahydrobiopterin

OOrphanetics Pharma Entwicklungs GmbH

Phase 1

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

Timeline

Expected Milestones

Q1 2031

Phase 3Recruiting

Phenylketonuria

NCT06302348

Clinical

Trials by Indication[1]

Phenylketonuria1 trial

A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

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

Regulatory

FDA Approval Timeline[4]

Jul 28, 2025

SEPHIENCE

NDA NDA219666

Sources

References

[1]
ClinicalTrials.gov — 1 trial record 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

Q1 2031

Trials by Indication[1]

Phenylketonuria1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

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