Sapropterin free base

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MedKoo CAT#: 318853

CAS#: 62989-33-7 (free base)

Description: Sapropterin, also known as Tetrahydrobiopterin (BH4, THB, trade name Kuvan) or sapropterin (INN) is a naturally occurring essential cofactor of the three aromatic amino acid hydroxylase enzymes, used in the degradation of amino acid phenylalanine and in the biosynthesis of the neurotransmitters serotonin (5-hydroxytryptamine, 5-HT), melatonin, dopamine, norepinephrine (noradrenaline), epinephrine (adrenaline), and is a cofactor for the production of nitric oxide (NO) by the nitric oxide synthases. Chemically, its structure is that of a reduced pteridine derivative.

Chemical Structure

Sapropterin free base
CAS# 62989-33-7 (free base)

Theoretical Analysis

MedKoo Cat#: 318853
Name: Sapropterin free base
CAS#: 62989-33-7 (free base)
Chemical Formula: C9H15N5O3
Exact Mass: 241.11749
Molecular Weight: 241.251
Elemental Analysis: C, 44.81; H, 6.27; N, 29.03; O, 19.90

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Related CAS #: 62989-33-7 (free base)   69056-38-8 (HCl)   90366-07-7 (sesqui HCl)   17528-72-2 (free base)  

Synonym: Tetrahydro-6-biopterin; Dapropterin; Phenoptin; THB; BPH4; 6R-BH4; Tetrahydrobiopterin, sapropterin; trade name Kuvan.

IUPAC/Chemical Name: (6R)-2-Amino-6-[(1R,2S)-1,2-dihydroxypropyl]-5,6,7,8-tetrahydropteridin-4(1H)-one


InChi Code: InChI=1S/C9H15N5O3/c1-3(15)6(16)4-2-11-7-5(12-4)8(17)14-9(10)13-7/h3-4,6,12,15-16H,2H2,1H3,(H4,10,11,13,14,17)/t3-,4+,6-/m0/s1

SMILES Code: O=C1N=C(N)NC2=C1N[C@@H]([C@@H](O)[C@@H](O)C)CN2

Appearance: Solid powder

Purity: >98% (or refer to the Certificate of Analysis)

Shipping Condition: Shipped under ambient temperature as non-hazardous chemical. This product is stable enough for a few weeks during ordinary shipping and time spent in Customs.

Storage Condition: Dry, dark and at 0 - 4 C for short term (days to weeks) or -20 C for long term (months to years).

Solubility: Soluble in DMSO, not in water

Shelf Life: >2 years if stored properly

Drug Formulation: This drug may be formulated in DMSO

Stock Solution Storage: 0 - 4 C for short term (days to weeks), or -20 C for long term (months).

HS Tariff Code: 2934.99.9001

Preparing Stock Solutions

The following data is based on the product molecular weight 241.251 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.

Recalculate based on batch purity %
Concentration / Solvent Volume / Mass 1 mg 5 mg 10 mg
1 mM 1.15 mL 5.76 mL 11.51 mL
5 mM 0.23 mL 1.15 mL 2.3 mL
10 mM 0.12 mL 0.58 mL 1.15 mL
50 mM 0.02 mL 0.12 mL 0.23 mL

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1: Doyle S, O'Regan M, Stenson C, Bracken J, Hendroff U, Agasarova A, Deverell D, Treacy EP. Extended Experience of Lower Dose Sapropterin in Irish Adults with Mild Phenylketonuria. JIMD Rep. 2017 Oct 14. doi: 10.1007/8904_2017_63. [Epub ahead of print] PubMed PMID: 29030855.

2: Kör D, Yılmaz BŞ, Bulut FD, Ceylaner S, Mungan NÖ. Improved metabolic control in tetrahydrobiopterin (BH4), responsive phenylketonuria with sapropterin administered in two divided doses vs. a single daily dose. J Pediatr Endocrinol Metab. 2017 Jul 26;30(7):713-718. doi: 10.1515/jpem-2016-0461. PubMed PMID: 28593914.

3: Muntau AC, Burlina A, Eyskens F, Freisinger P, De Laet C, Leuzzi V, Rutsch F, Sivri HS, Vijay S, Bal MO, Gramer G, Pazdírková R, Cleary M, Lotz-Havla AS, Munafo A, Mould DR, Moreau-Stucker F, Rogoff D. Efficacy, safety and population pharmacokinetics of sapropterin in PKU patients <4 years: results from the SPARK open-label, multicentre, randomized phase IIIb trial. Orphanet J Rare Dis. 2017 Mar 9;12(1):47. doi: 10.1186/s13023-017-0600-x. PubMed PMID: 28274234; PubMed Central PMCID: PMC5343543.

4: Hawks Z, Shimony J, Rutlin J, Grange DK, Christ SE, White DA. Pretreatment cognitive and neural differences between sapropterin dihydrochloride responders and non-responders with phenylketonuria. Mol Genet Metab Rep. 2017 Feb 23;12:8-13. doi: 10.1016/j.ymgmr.2017.01.013. eCollection 2017 Sep. PubMed PMID: 28271047; PubMed Central PMCID: PMC5323508.

5: Feldmann R, Wolfgart E, Weglage J, Rutsch F. Sapropterin treatment does not enhance the health-related quality of life of patients with phenylketonuria and their parents. Acta Paediatr. 2017 Jun;106(6):953-959. doi: 10.1111/apa.13799. Epub 2017 Mar 19. PubMed PMID: 28235150.

6: Yildiz Y, Dursun A, Tokatli A, Coskun T, Sivri S. Partial hydatidiform mole in a phenylketonuria patient treated with sapropterin dihydrochloride. Gynecol Endocrinol. 2017 Jan;33(1):19-20. doi: 10.1080/09513590.2016.1247796. Epub 2016 Nov 29. PubMed PMID: 27898272.

7: Fluegge K. A reply to 'Metabolic effects of sapropterin treatment in autism spectrum disorder: a preliminary study'. Transl Psychiatry. 2016 Apr 26;6:e793. doi: 10.1038/tp.2016.24. PubMed PMID: 27115124; PubMed Central PMCID: PMC4872393.

8: Tansek MZ, Groselj U, Kelvisar M, Kobe H, Lampret BR, Battelino T. Long-term BH4 (sapropterin) treatment of children with hyperphenylalaninemia - effect on median Phe/Tyr ratios. J Pediatr Endocrinol Metab. 2016 May 1;29(5):561-6. doi: 10.1515/jpem-2015-0337. PubMed PMID: 26910740.

9: Ünal Ö, Gökmen-Özel H, Coşkun T, Özgül RK, Yücel D, Hişmi B, Tokatlı A, Dursun A, Sivri HS. Sapropterin dihydrochloride treatment in Turkish hyperphenylalaninemic patients under age four. Turk J Pediatr. 2015 May-Jun;57(3):213-8. PubMed PMID: 26701937.

10: Winn SR, Scherer T, Thöny B, Harding CO. High dose sapropterin dihydrochloride therapy improves monoamine neurotransmitter turnover in murine phenylketonuria (PKU). Mol Genet Metab. 2016 Jan;117(1):5-11. doi: 10.1016/j.ymgme.2015.11.012. Epub 2015 Nov 26. PubMed PMID: 26653793; PubMed Central PMCID: PMC4706464.

11: Reverter E, Mesonero F, Seijo S, Martínez J, Abraldes JG, Peñas B, Berzigotti A, Deulofeu R, Bosch J, Albillos A, García-Pagán JC. Effects of Sapropterin on Portal and Systemic Hemodynamics in Patients With Cirrhosis and Portal Hypertension: A Bicentric Double-Blind Placebo-Controlled Study. Am J Gastroenterol. 2015 Jul;110(7):985-92. doi: 10.1038/ajg.2015.185. Epub 2015 Jun 16. PubMed PMID: 26077176.

12: Trefz FK, Muntau AC, Lagler FB, Moreau F, Alm J, Burlina A, Rutsch F, Bélanger-Quintana A, Feillet F; KAMPER investigators. The Kuvan(®) Adult Maternal Paediatric European Registry (KAMPER) Multinational Observational Study: Baseline and 1-Year Data in Phenylketonuria Patients Responsive to Sapropterin. JIMD Rep. 2015;23:35-43. doi: 10.1007/8904_2015_425. Epub 2015 Mar 31. PubMed PMID: 25822821; PubMed Central PMCID: PMC4484899.

13: Somaraju UR, Merrin M. Sapropterin dihydrochloride for phenylketonuria. Cochrane Database Syst Rev. 2015 Mar 27;(3):CD008005. doi: 10.1002/14651858.CD008005.pub4. Review. PubMed PMID: 25812600.

14: Longo N, Arnold GL, Pridjian G, Enns GM, Ficicioglu C, Parker S, Cohen-Pfeffer JL; Phenylketonuria Demographics, Outcomes and Safety Registry. Long-term safety and efficacy of sapropterin: the PKUDOS registry experience. Mol Genet Metab. 2015 Apr;114(4):557-63. doi: 10.1016/j.ymgme.2015.02.003. Epub 2015 Feb 16. PubMed PMID: 25724073.

15: Bushueva TV, Kuzenkova LM, Borovik TÉ, Nazarenko LP, Seitova GN, Filimonova MN, Pichkur NA, Samonenko NV, Shkurko TA, Akhmadeeva ÉN, Mardanova AK, Garifullina ÉR, Kovtun OP, Bazhenova IuL, Alimova IL, Kostiakova EA, Minaĭcheva LI, Saliukova OA, Sivokha VM, Rozenson OL. [Open, non-comparative phase III clinical study to evaluate the efficacy and safety of sapropterin in patients with phenylketonuria and hyperphenylalaninemia]. Vestn Ross Akad Med Nauk. 2014;(7-8):69-77. Russian. PubMed PMID: 25563006.

16: Burton B, Grant M, Feigenbaum A, Singh R, Hendren R, Siriwardena K, Phillips J 3rd, Sanchez-Valle A, Waisbren S, Gillis J, Prasad S, Merilainen M, Lang W, Zhang C, Yu S, Stahl S. A randomized, placebo-controlled, double-blind study of sapropterin to treat ADHD symptoms and executive function impairment in children and adults with sapropterin-responsive phenylketonuria. Mol Genet Metab. 2015 Mar;114(3):415-24. doi: 10.1016/j.ymgme.2014.11.011. Epub 2014 Nov 26. PubMed PMID: 25533024.

17: Stockler-Ipsiroglu S, Yuskiv N, Salvarinova R, Apatean D, Ho G, Cheng B, Giezen A, Lillquist Y, Ueda K. Individualized long-term outcomes in blood phenylalanine concentrations and dietary phenylalanine tolerance in 11 patients with primary phenylalanine hydroxylase (PAH) deficiency treated with Sapropterin-dihydrochloride. Mol Genet Metab. 2015 Mar;114(3):409-14. doi: 10.1016/j.ymgme.2014.11.014. Epub 2014 Dec 3. PubMed PMID: 25497838.

18: Jurecki ER, Cunningham A, Mahoney JJ, Tingley D, Chung S, James N, Cohen-Pfeffer JL. Sapropterin Dihydrochloride Mixed With Common Foods and Beverages. Top Clin Nutr. 2014 Oct;29(4):325-331. PubMed PMID: 25382934; PubMed Central PMCID: PMC4213132.

19: Qi Y, Mould DR, Zhou H, Merilainen M, Musson DG. A prospective population pharmacokinetic analysis of sapropterin dihydrochloride in infants and young children with phenylketonuria. Clin Pharmacokinet. 2015 Feb;54(2):195-207. doi: 10.1007/s40262-014-0196-4. PubMed PMID: 25338975; PubMed Central PMCID: PMC4306193.

20: Longo N, Siriwardena K, Feigenbaum A, Dimmock D, Burton BK, Stockler S, Waisbren S, Lang W, Jurecki E, Zhang C, Prasad S. Long-term developmental progression in infants and young children taking sapropterin for phenylketonuria: a two-year analysis of safety and efficacy. Genet Med. 2015 May;17(5):365-73. doi: 10.1038/gim.2014.109. Epub 2014 Sep 18. PubMed PMID: 25232857.

Additional Information

Related CAS#
69056-38-8 (Sapropterin dihydrochloride)
62989-33-7 (Sapropterin)