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 7 indications[4].[1]
Kuvan is one of 78 small molecule programs targeting Gastroparesis. It is currently in Approved.
7
Indications
23
Total Trials
3
Active Trials
77
Competitors
2
FDA Applications
NDA/BLA/ANDA regulatory filings
Pipeline
Status by Indication[4][1]
Indication
Competition
Competitive Landscape
55 competitors in Autism Spectrum Disorder
#DrugCompanyPhaseTrials
1Kuvan
BioMarin PharmaceuticalApproved
Buspar
Bristol-Myers SquibbApproved
Namenda
AbbVieApproved
Lioresal
NovartisApproved
Marinol
Jazz PharmaceuticalsApproved
Aricept
EisaiApproved
Epidiolex
Jazz PharmaceuticalsApproved
Abilify
OtsukaApproved
Neurontin
VViatris Specialty LLC
Approved
Strattera
MMap77
Approved
Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.
Timeline
Expected Milestones
Clinical
Trials by Indication[1]
Phenylketonuria6 trials
Adult Subject1 trial
Sickle Cell Disease1 trial
Executive Function1 trial
12 more indications
Endothelial Dysfunction1 trial
Peripheral Artery Disease1 trial
Head and Neck Squamous Cell Carcinoma1 trial
LAL1 trial
Tolerance1 trial
Peripheral Arterial Disease1 trial
Neurocognitive Function1 trial
Cognitive Developmental Disorder1 trial
Regulatory
FDA Approval Timeline[5]
Dec 13, 2007
KUVAN
NDA NDA022181
Dec 19, 2013
KUVAN
NDA NDA205065
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]
- [6]
Details
Drug Info
Quick Facts
- Mechanism of Action
- Phenylalanine-4-Hydroxylase Activator[3]
- Modality
- Small molecule[3]
- Highest Phase
- Approved[1]
- Company