Sapropterin
Clinical data
Trade namesKuvan, Biopten, other
Other namesTetrahydrobiopterin, Sapropterin hydrochloride (JAN JP), Sapropterin dihydrochloride (USAN US)
AHFS/Drugs.comMonograph
MedlinePlusa608020
License data
Pregnancy
category
Routes of
administration
By mouth
Legal status
Legal status
Pharmacokinetic data
Elimination half-life4 hours (healthy adults)
6–7 hours (PKU patients)
Duration of action24 hours[2]
Identifiers
  • (6R)-2-Amino-6-[(1R,2S)-1,2-dihydroxypropyl]-5,6,7,8-tetrahydropteridin-4(1H)-one
Chemical and physical data
FormulaC9H15N5O3
Molar mass241.251 g·mol−1
3D model (JSmol)
  • CC(C(C1CNC2=C(N1)C(=O)N=C(N2)N)O)O
  • 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 checkY
  • Key:FNKQXYHWGSIFBK-RPDRRWSUSA-N checkY
 ☒NcheckY (what is this?)  (verify)

Sapropterin, also known as tetrahydrobiopterin (BH4, THB), is a medication used to treat phenylketonuria or tetrahydrobiopterin deficiency.[2][3] It is used together with dietary changes.[2] Levels of phenylalanine in the blood are measured during the first month to determine if it is effective.[2] It is taken by mouth.[2]

Common side effects include headache and runny nose.[3] Other side effects may include anaphylaxis, stomach inflammation, and hyperactivity.[2] Use may be considered in pregnancy if diet alone does not control phenylalanine levels.[5] It is a cofactor which improves the activity of phenylalanine hydroxylase (PAH), which breaks down phenylalanine.[2]

Sapropterin was approved for medical use in the United States in 2007,[2] Europe in 2008,[3] and Canada in 2010.[6] In Canada it costs about 48,000 to 168,000 CAD a year for a 68 kg person in 2017.[4] In the United States this amount costs 38,000 to 152,000 USD per year while in the United Kingdom it is about £25,000 to £100,000 as of 2021.[7][8] It is sold under the brand names Kuvan and Biopten.[9]

References

edit
  1. ^ a b "Sapropterin (Kuvan) Use During Pregnancy". Drugs.com. 17 May 2019. Archived from the original on 29 October 2020. Retrieved 4 March 2020.
  2. ^ a b c d e f g h i j "Sapropterin Monograph for Professionals". Drugs.com. Archived from the original on 29 August 2021. Retrieved 10 October 2021.
  3. ^ a b c d e "Kuvan". Archived from the original on 14 August 2020. Retrieved 10 October 2021.
  4. ^ a b "Sapropterin dihydrochloride (Kuvan)" (PDF). CADTH. September 2017. Archived (PDF) from the original on 11 February 2020. Retrieved 10 October 2021.
  5. ^ "Sapropterin (Kuvan) Use During Pregnancy". Drugs.com. Archived from the original on 29 October 2020. Retrieved 10 October 2021.
  6. ^ Patient Group Input Submissions: sapropterin dihydrochloride (Kuvan) for Phenylketonuria (PKU). Canadian Agency for Drugs and Technologies in Health. 2017. Archived from the original on 12 October 2021. Retrieved 10 October 2021.
  7. ^ "Sapropterin Prices, Coupons & Patient Assistance Programs". Drugs.com. Archived from the original on 29 August 2021. Retrieved 10 October 2021.
  8. ^ BNF (80 ed.). BMJ Group and the Pharmaceutical Press. September 2020 – March 2021. p. 1139. ISBN 978-0-85711-369-6.{{cite book}}: CS1 maint: date format (link)
  9. ^ "Australian Public Assessment Report for Sapropterin dihydrochloride" (PDF). Archived (PDF) from the original on 12 October 2021. Retrieved 10 October 2021.