Sapropterin Hydrochloride

(rINNM)
Synonyms: Dapropterin Hydrochloride; Hidrocloruro de sapropterina; Sapropterin Dihydrochloride de; Sapropterini Hydrochloridum; SUN-0588 (sapropterin or sapropterin hydrochloride); T-1401; (6R)-5,6,7,8-Tetrahydrobiopterin Hydrochloride. (−)-(6R)-2-Amino-6-[(1R,2S)-1,2-dihydroxypropyl]-5,6,7,8-tetrahydro-4(3H)-pteridinone dihydrochloride.
Cyrillic synonym: Сапроптерина Гидрохлорид.

💊 Chemical information

Chemical formula: C9H15N5O3,2HCl = 314.2.
CAS — 62989-33-7 (sapropterin); 69056-38-8 (sapropterin hydrochloride).
ATC — A16AX07.
ATC Vet — QA16AX07.

💊 Profile

Sapropterin, the active form of tetrahydrobiopterin, is used as the hydrochloride for the diagnosis and treatment of variant forms of phenylketonuria associated with tetrahydrobiopterin (BH4) deficiency. It is given by mouth in daily doses of 1 to 20 mg/kg, in 2 or more divided doses. It has also been tried for the correction of hyperphenylalaninaemia seen in patients receiving treatment for leukaemia and which has been suggested as a possible cause of neurological symptoms.
1. Ueda S, et al. Tetrahydrobiopterin restores endothelial function in long-term smokers. J Am Coll Cardiol 2000; 35: 71–5
2. Thony B, et al. Tetrahydrobiopterin biosynthesis, regeneration and functions. Biochem J 2000; 347: 1–16
3. Muntau AC, et al. Tetrahydrobiopterin as an alternative treatment for mild phenylketonuria. N Engl J Med 2002; 347: 2122–32
4. Okano Y, et al. In vivo studies of phenylalanine hydroxylase by phenylalanine breath test: diagnosis of tetrahydrobiopterin-responsive phenylalanine hydroxylase deficiency. Pediatr Res 2004; 56: 714–19
5. Pérez-Dueñas B, et al. Tetrahydrobiopterin responsiveness in patients with phenylketonuria. Clin Biochem 2004; 37: 1083–90
6. Opladen T, et al. Severe mucitis after sublingual administration of tetrahydrobiopterin in a patient with tetrahydrobiopterin-responsive phenylketonuria. Eur J Pediatr 2005; 164: 395–6
7. Fiege B, Blau N. Assessment of tetrahydrobiopterin (BH ) responsiveness in phenylketonuria. J Pediatr 2007; 150: 627–30
8. Levy HL, et al. Sapropterin Research Group. Efficacy of sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase III randomised placebo-controlled study. Lancet 2007; 370: 504–10
9. Cosentino F, et al. Chronic treatment with tetrahydrobiopterin reverses endothelial dysfunction and oxidative stress in hypercholesterolaemia. Heart 2008; 94: 487–92.

💊 Preparations

Proprietary Preparations

Jpn: Biopten; USA: Kuvan.
Published May 08, 2019.