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Kuvan

(Sapropterin)

Phase 2Small molecule

BioMarin Pharmaceutical

Kuvan is one of 96 small molecule programs targeting Tic Disorder. It is currently in Phase 2.

4

Indications

10

Total Trials

0

Active Trials

95

Competitors

2

FDA Applications

NDA/BLA/ANDA regulatory filings

6

Patents

Pipeline

Status by Indication[3][1]

Indication

Tic DisorderActive, not recruiting

Portal HypertensionActive, not recruiting

HyperphenylalaninemiaActive, not recruiting

PhenylketonuriaActive, not recruiting

Tic DisorderPhase 2

Active, not recruiting

Portal HypertensionPhase 2/Phase 3

Active, not recruiting

HyperphenylalaninemiaPreclinical

Active, not recruiting

PhenylketonuriaPreclinical

Active, not recruiting

Competition

Competitive Landscape

71 competitors in Tic Disorder

#DrugCompanyPhaseTrials

Lithobid

SSolvay Pharmaceuticals

Buspar

Bristol-Myers Squibb

Abilify

Lynparza

Vitrakvi

Retevmo

Balversa

Johnson & Johnson

Ibrance

Haldol

Rituxan

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

Clinical

Trials by Indication[1]

Phenylketonuria5 trials

Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria

Terminated· BioMarin Pharmaceutical logo

BioMarin Pharmaceutical·Phase 2·NCT01977820·2 enrolled

Fluorodeoxyglucose Positron Emission Tomography (FDG PET) Findings in Patients With Phenylketonuria Before and After KUVAN Therapy

Completed·—·N/A·NCT00986973·6 enrolled

The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)

Completed·UUniversity of Missouri-Columbia··NCT00964236·20 enrolled

Show 2 more trials

Sapropterin in Individuals With Phenylketonuria

Completed·WWashington University School of Medicine··NCT00730080·45 enrolled

Nutritional Status in Phenylketonuria

Completed·UUniversidade do Porto··NCT03820804·94 enrolled

Treatment1 trial

Study of Kuvan Treatment in Adults With GTPCH Deficiency

Completed·UUniversity of Utah·Phase 1/2·NCT01425528·12 enrolled

Trial1 trial

Trial of Kuvan in Lesch-Nyhan Disease

Withdrawn·UUniversity of California, San Diego·Phase 2/3·NCT00935753

Hyperphenylalaninemia1 trial

Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy

Terminated·TThe University of Texas Health Science Center, Houston·N/A·NCT01541397·6 enrolled

Portal Hypertension1 trial

Randomized Controlled Trial to Assess the Effects of Sapropterin on Hepatic and Systemic Hemodynamics in Patients With Liver Cirrhosis and Portal Hypertension

Completed·FFundacion Clinic per a la Recerca Biomédica·Phase 2/3·NCT01456286·42 enrolled

1 more indication

Tic Disorder1 trial

Sapropterin as a Treatment for Autistic Disorder

Completed·TThe Children's Health Council·Phase 2·NCT00850070·46 enrolled

Regulatory

FDA Approval Timeline[4]

Dec 13, 2007

KUVAN

NDA NDA022181

Dec 19, 2013

KUVAN

NDA NDA205065

Sources

References

[1]
ClinicalTrials.gov — 10 trial records for Sapropterin. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Sapropterin compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Sapropterin. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA205065). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Kuvan. U.S. Food & Drug Administration.
[6]
SEC EDGAR — BioMarin Pharmaceutical company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 2[1]
Company: BioMarin Pharmaceutical

Patent Landscape[5]

Patent Cliff

Nov 1, 2032

Earliest patent expiration — generic entry possible after this date

Patent #Expires

7566462Nov 16, 2025

8003126Nov 16, 2025

7566462*PEDMay 16, 2026

8003126*PEDMay 16, 2026

9216178Nov 1, 2032

1 more patents

9216178*PEDMay 1, 2033

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