How many drugs treat Classical phenylketonuria?

Drug Landscape tracks 3 marketed treatments for Classical phenylketonuria (deduplicated by molecule). 0 Phase 3 candidates and 0 Phase 2 candidates are in the pipeline.

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Last reviewed 2026-06-10 · How we verify

Classical phenylketonuria — Treatment Landscape & Competitive Intelligence

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Classical phenylketonuria (disease) competitive landscape: 3 marketed treatments tracked, 0 Phase 3 candidates, 0 Phase 2 candidates. Recent regulatory actions and upcoming PDUFA dates across the entire treatment set.

3 marketed 0 Phase 3 0 Phase 2 Live · 30-min refresh

Marketed treatment landscape

Approved drugs treating Classical phenylketonuria, deduplicated by molecule. See the full disease page for line-of-therapy detail.

Drug	Generic	Sponsor	Class	Target	First approval
Kuvan	SAPROPTERIN	BioMarin	Phenylalanine Hydroxylase Activator	Nitric oxide synthase, brain	2007-01-01
Palynziq	Palynziq	Emory University
Tetrahydrobiopterin	Tetrahydrobiopterin	University of Massachusetts, Worcester		Nitric oxide synthase, brain, Phenylalanine-4-hydroxylase

Phase 3 pipeline

No Phase 3 pipeline candidates tracked.

Phase 2 pipeline

No Phase 2 pipeline candidates tracked.

Recent regulatory actions (last 90 days)

No regulatory actions in the last 90 days for this treatment set.

Upcoming PDUFA dates (next 180 days)

No PDUFA dates in the next 180 days for this treatment set.

Sponsor landscape

BioMarin · 1 drug in Classical phenylketonuria
Emory University · 1 drug in Classical phenylketonuria
University of Massachusetts, Worcester · 1 drug in Classical phenylketonuria

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Cite this brief

Drug Landscape (2026). Classical phenylketonuria — Treatment Landscape & Competitive Intelligence Brief. https://druglandscape.com/ci/disease/classical-phenylketonuria. Accessed 2026-06-10.

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Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing