Sapropterin

Overview

Sapropterin (tetrahydrobiopterin or BH4) is a cofactor in the synthesis of nitric oxide. It is also essential in the conversion of phenylalanine to tyrosine by the enzyme phenylalanine-4-hydroxylase; the conversion of tyrosine to L-dopa by the enzyme tyrosine hydroxylase; and conversion of tryptophan to 5-hydroxytryptophan via tryptophan hydroxylase.

Indication

For the treatment of tetrahydrobiopterin (BH4) deficiency.

Associated Conditions

Hyperphenylalaninemia

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
Study on Sapropterin Dihydrochloride Oral Suspension in Healthy Subjects	2024/07/01	NCT06481709	Phase 1	Completed	APR Applied Pharma Research s.a.
PD-1 Antibody and Sapropterin Dihydrochloride in Patients With PDAC	2024/05/02	NCT06396637	Phase 2	Recruiting	Sun Yat-sen University
BH4 in the Prevention and Treatment of Radiation-induced Skin Reactions for Head and Neck Squamous Cell Carcinoma (Including Nasopharyngeal Carcinoma)	2022/03/28	NCT05299203	Phase 1	UNKNOWN	West China Hospital
A Study for Assessing the Efficacy and Safety of BH4 in Radiation Enteritis	2021/12/01	NCT05138887	Phase 2	UNKNOWN	West China Second University Hospital
A Study for Assessing the Efficacy and Safety of Tetrahydrobiopterin in Radiation-Induced Skin Injury	2021/11/09	NCT05114226	Phase 1	UNKNOWN	West China Second University Hospital
The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication	2021/03/16	NCT04800692	Phase 1	Recruiting	Louis Messina
L-arginine Study for Persistent Symptoms of Schizophrenia	2019/08/13	NCT04054973	Phase 2	Withdrawn	University of Massachusetts, Worcester
O2 Transport and Utilization in Health and Lung Disease	2019/07/10	NCT04014712	Phase 1	Withdrawn	University of Massachusetts, Amherst
Retrospective Observational Safety Effectiveness With Kuvan in hpA	2019/03/06	NCT03864029	N/A	Completed	BioMarin Pharmaceutical
Nutritional Status in Phenylketonuria	2019/01/29	NCT03820804	N/A	Completed	Universidade do Porto

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
SAPROPTERIN DIHYDROCHLORIDE	Par Pharmaceutical, Inc.	49884-873	ORAL	500 mg in 1 1	11/18/2020
JAVYGTOR	Dr. Reddys Laboratories Inc	43598-097	ORAL	100 mg in 1 1	1/30/2022
SAPROPTERIN DIHYDROCHLORIDE	Par Pharmaceutical, Inc.	49884-948	ORAL	100 mg in 1 1	4/14/2020
JAVYGTOR	Dr. Reddys Laboratories Inc	43598-162	ORAL	500 mg in 1 1	11/3/2022
Sapropterin dihydrochloride	NorthStar RxLLC	72603-194	ORAL	500 mg in 1 1	12/4/2023
sapropterin dihydrochloride	NorthStar RxLLC	72603-191	ORAL	100 mg in 1 1	12/4/2023
Sapropterin Dihydrochloride	Dr. Reddys Laboratories Inc	43598-477	ORAL	100 mg in 1 1	12/8/2020
Kuvan	BioMarin Pharmaceutical Inc.	68135-482	ORAL	500 mg in 1 1	2/8/2021
Sapropterin Dihydrochloride	Dr. Reddys Laboratories Inc.	43598-749	ORAL	100 mg in 1 1	12/20/2021
Sapropterin dihydrochloride	Camber Pharmaceuticals, Inc.	31722-047	ORAL	100 mg in 1 1	10/10/2022

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Kuvan	BioMarin International Limited,Shanbally,Ringaskiddy Cork Co.,Cork,Ireland	Authorised	12/2/2008
Sapropterin Dipharma	Dipharma Arzneimittel GmbH,Offheimer Weg 33,65549 Limburg a. d. Lahn,Germany	Authorised	2/16/2022

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
KUVAN sapropterin dihydrochloride 100 mg soluble tablet bottle	165738	BioMarin Pharmaceutical Australia Pty Ltd	Medicine	A	10/28/2010
KUVAN sapropterin dihydrochloride 500 mg oral powder sachet	297734	BioMarin Pharmaceutical Australia Pty Ltd	Medicine	A	12/13/2018
KUVAN sapropterin dihydrochloride 100 mg oral powder sachet	297728	BioMarin Pharmaceutical Australia Pty Ltd	Medicine	A	12/13/2018

Health Canada Drug Approvals

Approved Product	Company	DIN	Dosage Form	Strength	Market Date
KUVAN	Biomarin International Limited	02482207	Powder - Oral	100 MG / SACHET	12/5/2019
TEVA-SAPROPTERIN	teva canada limited	02547481	Tablet - Oral	100 MG	N/A
REDDY-SAPROPTERIN	dr reddy's laboratories ltd	02534533	Powder For Solution - Oral	100 MG / SACHET	12/11/2023
KUVAN	Biomarin International Limited	02350580	Tablet - Oral	100 MG	7/5/2010
REDDY-SAPROPTERIN	dr reddy's laboratories ltd	02535610	Powder For Solution - Oral	500 MG / SACHET	12/19/2023
KUVAN	Biomarin International Limited	02482215	Powder - Oral	500 MG / SACHET	12/5/2019

CIMA AEMPS Drug Approvals

Approved Product	Company	Registration Number	Pharmaceutical Form	Prescription Type	Status
KUVAN 100 mg, COMPRIMIDOS SOLUBLES	Biomarin International Limited	08481002	COMPRIMIDO	Uso Hospitalario	Commercialized

Philippines FDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Philippines FDA approvals found for this drug.

Saudi SFDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Saudi SFDA approvals found for this drug.

Malaysia NPRA Drug Approvals

Approved Product	Company	Registration Number	Dosage Form	Strength	Approval Date
No Malaysia NPRA approvals found for this drug.

UK EMC Drug Information

Medicine Name	MA Holder	MA Number	Pharmaceutical Form	Active Ingredient	Authorization Date
No UK EMC drug information found for this drug.

Related News

PTC Therapeutics' Sephience Receives Positive CHMP Opinion for Phenylketonuria Treatment Across All Age Groups

3 months ago

The European Medicines Agency's CHMP has issued a positive opinion for Sephience (sepiapterin) to treat phenylketonuria in patients of all ages and disease severities, addressing a significant unmet medical need.

Synlogic's Novel Probiotic Therapy Shows Promise in Phase 2 PKU Trial, Advancing to Phase 3

6 months ago

Synlogic's engineered probiotic SYNB1934 demonstrated a significant 34% reduction in phenylalanine levels in phenylketonuria patients during Phase 2 trials, outperforming its predecessor SYNB1618.

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