Drugs / sapropterin
sapropterin
also known as
sapropterin dihydrochloride · Kuvan · 6R-BH4 · BH4 · tetrahydrobiopterin · Phenoptin · RLF-OD032 · 6R-tetrahydrobiopterin
targets PAH via activation
Regulatory milestones approvals, filings & regulatory actions · 6 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed phenylketonuria 2 milestones | |||||
| Filed | EU (EMA) | Kuvan | phenylketonuria | 2007-11-08 | “submitted a Marketing Authorization Application (MAA) to the EMEA for sapropterin dihydrochloride as an oral treatment for patients suffering from HPA due to PKU” biomarin.com ↗ “has submitted the Marketing Authorization Application (MAA) to the European Medicines Agency (EMEA) for sapropterin dihydrochloride” biomarin.com ↗ |
| Filed | US (FDA) | Kuvan | phenylketonuria | 2007-05-24 | “submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Kuvan(TM) (sapropterin dihydrochloride)” biomarin.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansions (2) | US (FDA) | KUVAN | — | 2013-12-19 – 2014-04-23 | fda.gov ↗ |
| Approved | EU (EMA) | Kuvan | — | 2008-12-02 | europa.eu ↗ |
| Approved | US (FDA) | KUVAN | — | 2007-12-13 | fda.gov ↗ |
Trials 47
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 01 trial | ||||||
| Phase 0 | NCT02042196 | Dec 2013 → May 2016 | — | University of Colorado, Denver | Completed | No outcome recorded |
| Phase 15 trials | ||||||
| Phase 1 | NCT06987773 | Aug 2025 | — | APR Applied Pharma Research s.a. | Completed | No outcome recorded |
| Phase 1 | NCT06481709 | Jul → Aug 2024 | — | APR Applied Pharma Research s.a. | Completed | No outcome recorded |
| Phase 1 | NCT05299203 | May → Dec 2022 | head and neck squamous cell carcinoma | West China Hospital | Unknown | No outcome recorded |
| Phase 1 | NCT00789568 | Oct 2008 → Jan 2009 | phenylketonuria | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 1 | NCT00435331 | Mar → Sep 2008 | pulmonary arterial hypertension | Vanderbilt University | Completed | No outcome recorded |
| Phase 1/22 trials · 1 missed | ||||||
| Phase 1/2 | NCT01706965 | Oct 2012 → Jun 2016 | schizoaffective disorder, schizophrenia | New York State Psychiatric Institute | Completed | Missed primary |
| Phase 1/2 | NCT01425528 | Aug 2011 → Aug 2013 | GTP cyclohydrolase I deficiency | University of Utah | Completed | No outcome recorded |
| Phase 216 trials · 1 met primary · 3 missed | ||||||
| Phase 2 | NCT06396637 | Apr 2024 → Apr 2025 | pancreatic adenocarcinoma | Sun Yat-sen University | Unknown | No outcome recorded |
| Phase 2 | NCT04054973 | Sep 2019 → Dec 2021 | schizoaffective disorder, schizophrenia | University of Massachusetts, Worcester | Withdrawn | No outcome recorded |
| Phase 2 | NCT02947750 | Oct 2016 → Apr 2027 expected | chronic kidney disease | Emory University | Recruiting | Mixed |
| Phase 2 | NCT01977820 | Feb → Nov 2014 | phenylketonuria | BioMarin Pharmaceutical | Terminated | No outcome recorded Stop: Enrollment |
| Phase 2 | NCT01772758 | Aug 2011 → Jun 2016 | cystic fibrosis | Augusta University | Completed | No outcome recorded |
| Phase 2 | NCT01395394 | Jun 2011 → Jun 2013 | phenylketonuria | Emory University | Terminated | No outcome recorded Stop: Enrollment |
| Phase 2 | NCT00850070 | Mar 2009 → Aug 2011 | autism | The Children's Health Council | Completed | Missed primary |
| Phase 2 | NCT00841100 | Dec 2008 → Jul 2011 | phenylketonuria | University of Miami | Completed | No outcome recorded |
| Phase 2 | NCT00802893 | Dec 2008 → May 2009 | cardiovascular disorder | Johns Hopkins University | Terminated | No outcome recorded Stop: Enrollment |
| Phase 2 | NCT00625820 | May → Sep 2008 | chronic kidney disease, proteinuria | University of Michigan | Completed | Missed primary |
| Phase 2 | NCT00532844 | Sep 2007 → Nov 2008 | — | BioMarin Pharmaceutical | Completed | Met primary |
| Phase 2 | NCT00445978 | May 2007 → Aug 2008 | sickle cell disease | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 2 | NCT00403494 | Dec 2006 → Nov 2008 | intermittent vascular claudication | BioMarin Pharmaceutical | Completed | Missed primary |
| Phase 2 | NCT00355264 | Aug 2006 → Jun 2009 | hyperphenylalaninemia due to tetrahydrobiopterin deficiency | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 2 | NCT00325962 | May → Dec 2006 | hypertensive disorder | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 2 | NCT00104260 | Dec 2004 → Nov 2005 | phenylketonuria | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 2/33 trials · 1 missed | ||||||
| Phase 2/3 | NCT01456286 | Oct 2011 → Oct 2013 | cirrhosis of liver, portal hypertension | Fundacion Clinic per a la Recerca Biomédica | Completed | No outcome recorded |
| Phase 2/3 | NCT00943579 | Aug 2009 → Dec 2011 | autism | The Children's Health Council | Completed | Missed primary |
| Phase 2/3 | NCT00935753 | Apr 2009 → Feb 2010 | Lesch-Nyhan syndrome | University of California, San Diego | Withdrawn | No outcome recorded |
| Phase 38 trials · 3 met primary · 1 missed | ||||||
| Phase 3 | NCT01732471 | Nov 2012 → Oct 2013 | phenylketonuria | Merck KGaA, Darmstadt, Germany | Completed | No outcome recorded |
| Phase 3 | NCT01376908 | Jun 2011 → Jul 2014 | phenylketonuria | BioMarin Pharmaceutical | Completed | Met primary |
| Phase 3 | NCT01114737 | Aug 2010 → Mar 2013 | phenylketonuria | BioMarin Pharmaceutical | Completed | Missed primary |
| Phase 3 | NCT00838435 | Feb 2009 → Sep 2018 | phenylketonuria | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 3 | NCT00332189 | Jul 2006 → Aug 2009 | phenylketonuria | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 3 | NCT00272792 | Feb → Nov 2006 | phenylketonuria | BioMarin Pharmaceutical | Completed | Met primary |
| Phase 3 | NCT00225615 | Nov 2005 → Jun 2006 | phenylketonuria | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 3 | NCT00104247 | Mar 2005 → Feb 2006 | phenylketonuria | BioMarin Pharmaceutical | Completed | Met primary |
| Phase 44 trials | ||||||
| Phase 4 | NCT03493412 | Jul 2019 | peripheral arterial disease | University of Nebraska | Withdrawn | No outcome recorded Stop: Enrollment |
| Phase 4 | NCT01965912 | Oct 2013 → Jan 2023 | phenylketonuria | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 4 | NCT01617070 | May 2012 → Dec 2014 | phenylketonuria | University of Southern California | Completed | No outcome recorded |
| Phase 4 | NCT01082328 | May 2010 → May 2012 | phenylketonuria | Merck KGaA, Darmstadt, Germany | Completed | No outcome recorded |
| Phase not applicable7 trials · 1 missed | ||||||
| — | NCT02353312 | Mar 2015 → Dec 2018 | heart failure | Providence VA Medical Center | Completed | No outcome recorded |
| — | NCT01806051 | Mar 2013 → May 2014 | phenylketonuria | Dr. Linda Randolph | Terminated | No outcome recorded |
| — | NCT01541397 | Jun 2011 → Aug 2012 | phenylketonuria | The University of Texas Health Science Center, Houston | Terminated | No outcome recorded |
| — | NCT01412437 | Apr 2011 → Mar 2012 | phenylketonuria | Children's National Research Institute | Withdrawn | No outcome recorded Stop: Enrollment |
| — | NCT01141595 | Jul 2010 → Sep 2011 | autism spectrum disorder | The University of Texas Health Science Center, Houston | Completed | No outcome recorded |
| — | NCT01135186 | May 2010 → Nov 2012 | gastroparesis | Stanford University | Completed | No outcome recorded |
| — | NCT00986973 | Mar 2010 → Jul 2011 | phenylketonuria | Children's Hospital of Philadelphia | Completed | Missed primary |
| Phase not stated1 trial | ||||||
| — | NCT00484991 | — | phenylketonuria | BioMarin Pharmaceutical | Approved for marketing | No outcome recorded |
| Comparator or background therapy2 trials | ||||||
| Phase 1 | NCT07255599 Comparator | Sep 2025 → Jan 2026 overdue | phenylketonuria | Sohag University | Active not recruiting | No outcome recorded |
| Phase 2 | NCT00423280 Comparator | Nov 2006 → Feb 2009 | coronary artery disorder | University of Oxford | Unknown | No outcome recorded |
News releases announcing trial results or a regulatory action · 27
| Date | Issuer | Release |
|---|---|---|
| 2015-03-30 | BioMarin Pharmaceutical | Results BioMarin Announces One Oral and 7 Poster Presentations at the 2015 American College of Medical Genetics and Genomics Annual Clinical Genetics Meeting biomarin.com ↗
There was a statistically significant difference between the baseline and week 13 ADHD-RS total score with Kuvan compared with placebo (p = 0.01). |
| 2014-09-24 | BioMarin Pharmaceutical | Regulatory FDA Receives Paragraph IV Notice Letter for KUVAN(R) (sapropterin dihydrochloride) Tablets biomarin.com ↗ |
| 2014-04-14 | BioMarin Pharmaceutical | Regulatory FDA Extends Market Exclusivity Six Months for BioMarin’s Rare Disease Therapy KUVAN(R) (sapropterin dihydrochloride) Powder for Oral Solution and Tablets biomarin.com ↗
The FDA action extends KUVAN's market exclusivity to June 2015 and is based on studies submitted in response to a written request by the FDA to investigate the use of KUVAN in pediatric patients from birth to age 6. |
| 2013-02-19 | BioMarin Pharmaceutical | Results BioMarin Announces Kuvan Significantly Improves Inattentiveness in Kuvan Responding PKU Patients biomarin.com ↗
Kuvan improved the ADHD-RS (p=0.085), driven by a statistically significant change in the inattention component of the score (p=0.036). |
| 2010-09-01 | BioMarin Pharmaceutical | Results Results From Investigator-Sponsored Trials Presented at SSIEM Conference biomarin.com ↗
The results of this exploratory study suggest improvements in executive abilities following treatment with sapropterin. |
| 2009-06-10 | BioMarin Pharmaceutical | Regulatory Kuvan Receives Priority Review Status From Health Canada biomarin.com ↗
BioMarin Pharmaceutical Inc. announced today that it has received notice of priority review status for Kuvan(R) (sapropterin dihydrochloride) as an oral treatment for patients with phenylketonuria (PKU) from Health Canada. |
| 2009-02-03 | BioMarin Pharmaceutical | Results Results From Phase 2 Clinical Study of 6R-BH4 in Peripheral Arterial Disease Not Statistically Significant biomarin.com ↗
There was no statistical significance observed between the 6R-BH4 treatment and placebo groups. |
| 2008-12-09 | BioMarin Pharmaceutical | Regulatory Kuvan Receives Marketing Approval in the European Union biomarin.com ↗
BioMarin will receive a milestone payment of $30 million for this marketing approval along with royalties on net sales of Kuvan in the E.U. |
| 2008-10-15 | BioMarin Pharmaceutical | Results BioMarin Announces Positive Results From Phase 2A Clinical Study of 6R-BH4 in Sickle Cell Disease biomarin.com ↗
Oral administration of 6R-BH4 was associated with an improvement in endothelial dysfunction in sickle cell disease patients. |
| 2008-09-25 | BioMarin Pharmaceutical | Regulatory Kuvan Receives Positive Opinion From CHMP for European Approval biomarin.com ↗ |
| 2008-07-16 | Daiichi Sankyo | Regulatory Asubio Pharma’s Inherited Metabolic Disease Agent Biopten®Receives Approval for Additional Indication daiichisankyo.com ↗
Asubio Pharma’s Inherited Metabolic Disease Agent Biopten®Receives Approval for Additional Indication |
| 2008-07-16 | BioMarin Pharmaceutical | Regulatory Biopten (Sapropterin Dihydrochloride) Approved by Japanese Ministry of Health for the Treatment of PKU biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that Asubio Pharma Co., Ltd. (a subsidiary of Daiichi Sankyo), has received marketing approval from the Japanese Ministry of Health, Labour and Welfare (MHLW) for a label extension of Biopten(R) (sapropterin dihydrochloride), which contains the same active ingredient as Kuvan(R) in the U.S., for the treatment of patients with phenylketonuria (PKU). |
| 2007-12-13 | BioMarin Pharmaceutical | Regulatory BioMarin Announces FDA Approval for Kuvan biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that the U.S. Food and Drug Administration (FDA) has granted marketing approval for Kuvan(TM) (sapropterin dihydrochloride) Tablets, the first specific drug therapy approved for the treatment of phenylketonuria (PKU). |
| 2007-11-08 | BioMarin Pharmaceutical | Regulatory MAA Submitted to EMEA for European Marketing Authorization of Sapropterin for Hyperphenylalaninemia (HPA) biomarin.com ↗ |
| 2007-11-01 | BioMarin Pharmaceutical | Regulatory BioMarin Announces Third Quarter 2007 Financial Results biomarin.com ↗
The FDA has assured us that the change of the action date is solely due to a staffing constraint, |
| 2007-10-17 | BioMarin Pharmaceutical | Results Kuvan, Naglazyme and Aldurazyme Data to Be Presented at the 57th Annual Meeting of the American Society of Human Genetics biomarin.com ↗
Mean daily Phe intake (dietary + supplement) increased from 16.8 to 43.8mg/kg/day with sapropterin (p<0.001) and from 16.3 to 23.5mg/kg/day with placebo (p=0.079). |
| 2007-08-13 | BioMarin Pharmaceutical | Results Positive Kuvan(TM) Pivotal Phase 3 Trial Results Published in The Lancet biomarin.com ↗
The study suggests that treatment with Kuvan results in significant reductions in blood Phe levels in some phenylketonuria (PKU) patients. |
| 2007-07-25 | BioMarin Pharmaceutical | Regulatory Kuvan Receives Priority Review Status from FDA biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing and granted priority review status to the New Drug Application (NDA) for Kuvan(TM) (sapropterin dihydrochloride). |
| 2007-05-24 | BioMarin Pharmaceutical | Regulatory BioMarin Submits New Drug Application for U.S. Marketing Authorization of Kuvan for Phenylketonuria (PKU) biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Kuvan(TM) (sapropterin dihydrochloride), an investigational oral small molecule for the treatment of patients with phenylketonuria (PKU) being developed in partnership with Merck Serono. |
| 2007-02-20 | BioMarin Pharmaceutical | Results BioMarin Announces Results From Phase 2 Clinical Study of 6R-BH4 in Poorly Controlled Hypertension biomarin.com ↗
Results demonstrate that there was no statistically significant or clinically meaningful effect of 6R-BH4 on any efficacy or safety parameter measured, relative to placebo. |
| 2007-01-16 | BioMarin Pharmaceutical | Results BioMarin Announces Positive Results From Phase 3 Diet Study of Phenoptin for PKU biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) today announced positive results from the Phase 3 diet study of Phenoptin(TM) (sapropterin dihydrochloride), in combination with diet, in 4-12 year old patients. |
| 2006-12-18 | BioMarin Pharmaceutical | Results BioMarin Announces Positive Results From Phase 3 Extension Study of Phenoptin for PKU biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) today announced positive results from the pivotal Phase 3 extension study of Phenoptin (sapropterin hydrochloride), an investigational oral small molecule for the treatment of patients with phenylketonuria (PKU), who have elevated phenylalanine (Phe) levels. |
| 2006-09-12 | BioMarin Pharmaceutical | Results BioMarin and Serono Announce Data on Phenoptin in PKU to be Presented at the 56th Annual Meeting of the American Society of Human Genetics biomarin.com ↗
The type and incidence of adverse events was similar in the Phenoptin and placebo groups. |
| 2006-03-15 | BioMarin Pharmaceutical | Results BioMarin and Serono Announce Positive Results From Phase 3 Clinical Study of Phenoptin for PKU biomarin.com ↗
data from the Phase 3 study demonstrate a statistically significant reduction at six weeks in blood phenylalanine (Phe) levels (p<0.0001) in patients receiving Phenoptin, compared with those receiving placebo. |
| 2006-01-25 | BioMarin Pharmaceutical | Regulatory BioMarin Receives FDA Fast Track Designation for Phenoptin for PKU biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for Phenoptin(TM) (sapropterin dihydrochloride) for phenylketonuria (PKU). |
| 2004-08-23 | BioMarin Pharmaceutical | Regulatory BioMarin Files Investigational New Drug Application for Phenoptin (6R-BH4) for the Treatment of PKU biomarin.com ↗
BioMarin Pharmaceutical Inc. announced that it has filed an investigational new drug application (IND) with the U.S. Food and Drug Administration (FDA) for Phenoptin(TM), BioMarin's proprietary oral formulation of 6R-BH4, for the treatment of phenylketonuria (PKU). |
| 2004-07-19 | BioMarin Pharmaceutical | Results BioMarin Announces Positive Results from Clinical Study of 6R-BH4 in PKU Patients biomarin.com ↗
BioMarin Pharmaceutical Inc. announced positive results from a pilot clinical study of 6R-BH4 in 20 patients with phenylketonuria (PKU). |
All press releases naming this drug 53 releases
Evidence & citations 12 cited values
Every value below carries the sentence it was read from. 52 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | sapropterin | “The primary objective of the study is to evaluate the proportion of responders (that is, greater than or equal to \[\>=\] 30 percent reduction from Baseline in blood...” NCT01082328 ↗22“The aim of the SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) trial was to assess the efficacy (improvement in daily phenylalanine tolerance, neuromotor...” PMID 28274234 ↗ Mar 2017 “Sapropterin dihydrochloride (subsequently referred to as sapropterin) (Kuvan®) was approved by the FDA for the treatment of hyperphenylalaninemia in 2007.” NCT00789568 ↗ “Kuvan® (sapropterin dihydrochloride) will be administered orally at a dose of 20 milligram per kilogram per day (mg/kg/day) once daily for 8 days.” NCT01732471 ↗ |
| Known as | 6R-BH4 | “Efficacy of sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase III randomised...” PMID 17693179 ↗ Aug 20076“The purpose of this study is to determine whether the addition of sapropterin dihydrochloride (6R-BH4) to existing treatment has any effect in patients with pulmonary arterial...” NCT00435331 ↗ |
| Known as | 6R-tetrahydrobiopterin | “6R-tetrahydrobiopterin (6R-BH4 or sapropterin dihydrochloride) is an investigational oral drug” NCT00625820 ↗ |
| Known as | BH4 | “Tetrahydrobiopterin（BH4）, also known as sapropterin, has been shown not only to reduce the severity of acute radiation dermatitis but also to enhance the repair of skin injury...” NCT05299203 ↗3“The FDA recently approved a new medication, Kuvan, a synthetic form of tetrahydrobiopterin (BH4) manufactured by BioMarin Pharmaceutical Inc., for patients with PKU.” NCT01617070 ↗ “Sapropterin Dihydrochloride (BH4, tetrahydrobiopterin)” NCT03493412 ↗ “Sapropterin Dihydrochloride (BH4, tetrahydrobiopterin)” NCT07255599 ↗ |
| Known as | Kuvan | “During the initial 26-week SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) study, addition of sapropterin dihydrochloride (Kuvan®; a synthetic formulation of the...” PMID 34344399 ↗ Aug 202112“A Pilot, Phase 1, Randomized, Open-Label, Single-Dose, Four-Way Crossover Study to Compare the Pharmacokinetics of Sapropterin Dihydrochloride 100 mg/mL Oral Suspension...” NCT06481709 ↗ “The primary objective of the study is to evaluate the proportion of responders (that is, greater than or equal to \[\>=\] 30 percent reduction from Baseline in blood...” NCT01082328 ↗ “Kuvan® (sapropterin dihydrochloride) will be administered orally at a dose of 20 milligram per kilogram per day (mg/kg/day) once daily for 8 days.” NCT01732471 ↗ |
| Known as | Phenoptin | “The primary objective is to evaluate the degree and frequency of response to Phenoptin™ (sapropterin dihydrochloride), as demonstrated by a reduction in blood phenylalanine...” NCT00104260 ↗ |
| Known as | RLF-OD032 | “A Pilot, Phase 1, Randomized, Open-Label, Single-Dose, Four-Way Crossover Study to Compare the Pharmacokinetics of Sapropterin Dihydrochloride 100 mg/mL Oral Suspension...” NCT06481709 ↗1“A) A single 10 mg/kg dose of a 100 mg/mL oral suspension of sapropterin dihydrochloride (RLF-OD032) , administered under fed conditions without water followed by B) A single 10...” NCT06987773 ↗ |
| Known as | sapropterin dihydrochloride | “The primary objective is to evaluate the degree and frequency of response to Phenoptin™ (sapropterin dihydrochloride), as demonstrated by a reduction in blood phenylalanine...” NCT00104260 ↗21“In each period, subjects will receive a single 10 mg/kg dose of sapropterin dihydrochloride on Day 1, under fasting or fed conditions, and with or without water, followed by 24...” NCT06481709 ↗ “Efficacy of sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase III randomised...” PMID 17693179 ↗ Aug 2007 “The sapropterin EAP (SEAP) is an open-label, multi-center program designed to provide access to sapropterin dihydrochloride for patients diagnosed with hyperphenylalaninemia due to PKU.” NCT00484991 ↗ “Sapropterin dihydrochloride (sapropterin, Kuvan®) is the only US- and EU-approved pharmaceutical version of naturally occurring 6R-BH(4), the cofactor required for PAH activity.” PMID 21646032 ↗ Mar 2011 “In each period, subjects will receive a single 10 mg/kg dose of sapropterin dihydrochloride on Day 1, under fed conditions, followed by 24 hours of PK sampling.” NCT06987773 ↗ “Kuvan® or sapropterin dihydrochloride is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4).” NCT01772758 ↗ “A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease” NCT00403494 ↗ “6R-tetrahydrobiopterin (6R-BH4 or sapropterin dihydrochloride) is an investigational oral drug” NCT00625820 ↗ “Sapropterin Dihydrochloride (BH4, tetrahydrobiopterin)” NCT03493412 ↗ “Sapropterin Dihydrochloride (BH4, tetrahydrobiopterin)” NCT07255599 ↗ |
| Known as | tetrahydrobiopterin | “Tetrahydrobiopterin（BH4）, also known as sapropterin, has been shown not only to reduce the severity of acute radiation dermatitis but also to enhance the repair of skin injury...” NCT05299203 ↗3“Efficacy of sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase III randomised...” PMID 17693179 ↗ Aug 2007 “Sapropterin Dihydrochloride (BH4, tetrahydrobiopterin)” NCT03493412 ↗ “Sapropterin Dihydrochloride (BH4, tetrahydrobiopterin)” NCT07255599 ↗ |
| Action | Activate | “Sapropterin enhances residual PAH activity in sapropterin-responsive PKU patients” PMID 21646032 ↗ Mar 2011 |
| Route | Oral | “We randomly assigned 42 patients to receive oral doses of sapropterin (10 mg/kg) and 47 patients to receive placebo, once daily for 6 weeks.” PMID 17693179 ↗ Aug 2007 |
| Target | PAH | “Sapropterin enhances residual PAH activity in sapropterin-responsive PKU patients” PMID 21646032 ↗ Mar 2011 |