Phenylketonuria

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At a Glance

Live from database

Live Alerts

PAN Foundation — Phenylketonuria status changedApply now ↗

Regulatory Watchboard

10 events
Feb 2026PALYNZIQ: New indication approved
FDAcompleted
Nov 2025PALYNZIQ: New indication approved
FDAcompleted
Oct 2025A Study to Evaluate the Safety and Efficacy of JNT-517 in Participants With Phenylketonuria (PKU)

Otsuka Pharmaceutical Development & Commercialization, Inc. — PHASE3

TrialRECRUITING
Sep 2025Evaluation of BH4 Responsiveness in Our PKU Patients

Sohag University — PHASE1

TrialACTIVE NOT RECRUITING
May 2025GMP Powdered Substitutes in PKU and TYR

Nutricia UK Ltd — NA

TrialRECRUITING
Apr 2025A Self-test Home-use Blood Phenylalanine Monitoring System Under the Brand Name Egoo Phe System Has Been Developed for Measurement of Phenylalanine (Phe) in Individuals Diagnosed With Phenylketonuria (PKU): The Study Purpose is to Evaluate Accuracy and Usability

Egoo Health Aps — NA

TrialENROLLING BY INVITATION
Feb 2025Effect of L-carnitine Supplementation on Phenylalanine and Brain-derived Neurotrophic Factor Levels in Infants and Children With Phenylketonuria

Mansoura University — PHASE4

TrialACTIVE NOT RECRUITING
Jan 2025Brain Aging in Phenylketonuria

Insel Gruppe AG, University Hospital Bern — NA

TrialRECRUITING
Jan 2025AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)

NGGT INC. — PHASE1

TrialRECRUITING
Oct 2024Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria

University of Missouri-Columbia — NA

TrialRECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

9 programs

FINANCIAL LANDSCAPE SUMMARY

9

Total programs

8

Open now

6

Foundation grants

3

Copay cards

Foundation Grants6

Good Days — Phenylketonuria

Good Days

OpenContact for detailsApply ↗

Patient Services Inc — Phenylketonuria

Patient Services Inc

OpenContact for detailsApply ↗

PAN Foundation — Phenylketonuria

PAN Foundation

ClosedContact for detailsApply ↗

NORD Patient Assistance — Phenylketonuria

NORD Patient Assistance

OpenContact for detailsApply ↗

HealthWell Foundation — Phenylketonuria

HealthWell Foundation

OpenContact for detailsApply ↗

The Assistance Fund — Phenylketonuria

The Assistance Fund

OpenContact for detailsApply ↗

Copay Assistance3

SAPROPTERIN DIHYDROCHLORIDE

BioMarin

OpenContact for detailsApply ↗

Palynziq

BioMarin

OpenContact for detailsApply ↗

Kuvan

BioMarin

OpenContact for detailsApply ↗

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Approved Treatments

4 FDA-approved

Sephience

(SEPIAPTERIN)Orphan drug

PTC Therapeutics Inc.

FDA label ↗

Palynziq

(PEGVALIASE-PQPZ)Orphan drug

BioMarin Pharmaceutical Inc.

FDA label ↗

Sapropterin Dihydrochloride

(SAPROPTERIN DIHYDROCHLORIDE)Orphan drug

Aurobindo Pharma Limited

FDA label ↗

Kuvan

(sapropterin)Orphan drugstandard

BioMarin Pharmaceutical, Inc.

12.1 Mechanism of Action KUVAN is a synthetic form of BH4, the cofactor for the enzyme phenylalanine hydroxylase (PAH). PAH hydroxylates Phe through a...

FDA label ↗

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Clinical Trial Landscape

20 active trials
4Phase 3
1Phase 4
5Phase 1
9N/A
1EARLY_PHASE1
20Total recruiting
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Recent News & Research

No recent news articles indexed yet for Phenylketonuria.
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Specialist Network

Top 6 by expertise

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