Kuvan
(Sapropterin Dihydrochloride)ApprovedSmall molecule
BioMarin PharmaceuticalSapropterin is used to treat phenylketonuria (PKU), a rare genetic disorder where the body cannot properly break down the amino acid phenylalanine. This medication helps lower phenylalanine levels in the blood by improving the body's ability to process it.[2] Kuvan is currently approved and in use across 17 indications[4].[1]
Kuvan is one of 469 small molecule programs targeting Coronary Artery Disease. It is currently in Approved.
17
Indications
22
Total Trials
2
Active Trials
468
Competitors
Pipeline
Status by Indication[4][1]
Indication
Showing 10 of 17 indications
Competition
Competitive Landscape
583 competitors in Schizophrenia
#DrugCompanyPhaseTrials
1Kuvan
BioMarin PharmaceuticalApproved
Inapsine
Alnylam PharmaceuticalsPreclinical
Zocor
OOrganon and Co
Preclinical
Ibrance
PfizerPreclinical
Rapamune
PPrism Pharmaceuticals
Preclinical
Persantine
Boehringer IngelheimPreclinical
Inversine
TTargacept Inc.
Preclinical
Permax
VValeant Pharmaceuticals
Preclinical
Orap
TevaPreclinical
Antidepressant
Johnson & JohnsonPreclinical
Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.
Timeline
Expected Milestones
Clinical
Trials by Indication[1]
Phenylketonuria5 trials
Peripheral Arterial Disease1 trial
Neurocognitive Function1 trial
12 more indications
Sickle Cell Disease1 trial
Executive Function1 trial
LAL1 trial
Adult Subject1 trial
Endothelial Dysfunction1 trial
Tolerance1 trial
Head and Neck Squamous Cell Carcinoma1 trial
Peripheral Artery Disease1 trial
Sources
References
- [1]
- [2]MedlinePlus Drug Information — Kuvan drug information. U.S. National Library of Medicine, National Institutes of Health.
- [3]
- [4]Open Targets Platform — Drug–disease associations for Sapropterin Dihydrochloride. EMBL-EBI & Wellcome Sanger Institute.
- [5]
Details
Drug Info
Quick Facts
- Mechanism of Action
- Phenylalanine-4-Hydroxylase Activator[3]
- Modality
- Small molecule[3]
- Highest Phase
- Approved[1]
- Company