NDAORALTABLETPriority Review
Approved
Dec 2007
Lifecycle
Post-LOE
Competitive Pressure
35/100
Clinical Trials
20
Data completeness
5/8 — Partial
Priority Review
KUVAN (sapropterin dihydrochloride) by BioMarin Pharmaceutical is bh4, the cofactor for the enzyme phenylalanine hydroxylase (pah). First approved in 2007.
Drug data last refreshed 2h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
KUVAN (sapropterin dihydrochloride) is an oral small-molecule cofactor for phenylalanine hydroxylase (PAH) approved in 2007 to treat phenylketonuria (PKU) by restoring residual PAH enzyme activity and reducing phenylalanine levels. It works by providing BH4, the essential cofactor that enables PAH to convert phenylalanine to tyrosine through normal metabolic oxidation.
Post-LOEPost-LOE
Post-LOE product with moderate competitive pressure (35/100) suggests stable but defending market share; brand teams focused on lifecycle management and patient retention rather than growth.
Mechanism of Action
BH4, the cofactor for the enzyme phenylalanine hydroxylase (PAH). PAH hydroxylates Phe through an oxidative reaction to form tyrosine. In patients with PKU, PAH activity is absent or deficient. Treatment with BH4 can activate residual PAH enzyme activity, improve the normal oxidative metabolism of…
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (20)
PD-1 Antibody and Sapropterin Dihydrochloride in Patients With PDAC
Started Apr 2024
20 enrolled
Pancreatic Adenocarcinoma Metastatic
The Effectiveness of Kuvan in Amish PKU Patients
Started Jan 2018
7 enrolled
Phenylketonuria
Retrospective Observational Safety Effectiveness With Kuvan in hpA
Started Oct 2017
26 enrolled
Tetrahydrobiopterin Deficiency
Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
Started Oct 2013
34 enrolled
Phenylketonuria
Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria
Started Nov 2012
90 enrolled
Phenylketonuria
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.