Sapropterin dihydrochloride
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Sapropterin dihydrochloride
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CAS No:
69056-38-8
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Formula:
C9H15N5O3.2ClH
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Chemical Name:
Sapropterin dihydrochloride
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Synonyms:
4(3H)-Pteridinone,2-amino-6-[(1R,2S)-1,2-dihydroxypropyl]-5,6,7,8-tetrahydro-,hydrochloride (1:2),(6R)-;4(1H)-Pteridinone,2-amino-6-(1,2-dihydroxypropyl)-5,6,7,8-tetrahydro-,dihydrochloride,[6R-[6R*(1R*,2S*)]]-;4(1H)-Pteridinone,2-amino-6-[(1R,2S)-1,2-dihydroxypropyl]-5,6,7,8-tetrahydro-,dihydrochloride,(6R)-;SUN 0588;(6β)-5,6,7,8-Tetrahydro-L-biopterin dihydrochloride;Biopten;Sapropterin dihydrochloride;Shiratori SN 0588;BioMarin T 1401;Kuvan;90687-77-7
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CAS No:
Description
Sapropterin dihydrochloride is a synthetic form of BH4 that is approved for the treatment of BH4 responsive PKU. (1) Sapropterin dihydrochloride can stimulate TH and TPH activities leading to improved dopamine and serotonin synthesis despite persistently elevated brain phenylalanine.(2) Sapropterin dihydrochloride is used to lower blood phenylalanine levels in tetrahydrobiopterin-responsive phenylketonuria in conjunction with a phenylalanine-restricted diet.
Sapropterin dihydrochloride is the dihydrochloride salt of sapropterin. It is used for the diagnosis and treatment of variant forms of phenylketonuria (hyperphenylalaninaemia) associated with tetrahydrobiopterin deficiency. Natural cofactor for phenylalanine hydroxylase, tyrosine hydroxylase, tryptophan hydroxylase, and nitric oxide synthetase. It has a role as a diagnostic agent and a coenzyme. It contains a sapropterin.
Sapropterin dihydrochloride Basic Attributes
314.17
313.070845
RG277LF5B3
DTXSID0040620
A16AX07
2933990090
Characteristics
132
0.76100
white crystalline solid
1.9±0.1 g/cm3
245-246 °C (decomp)
506.6ºC at 760 mmHg
260.2±32.9 °C
1.822
-20ºC
LD50 oral in rat: 1gm/kg
Safety Information
NONH for all modes of transport
3
R22
UO3516500
Xn
H302
|Warning|H315 (100%): Causes skin irritation [Warning Skin corrosion/irritation]|P261, P264, P271, P280, P302+P352, P304+P340, P305+P351+P338, P312, P321, P332+P313, P337+P313, P362, P403+P233, P405, and P501|Aggregated GHS information provided by 38 companies from 1 notifications to the ECHA C&L Inventory. Each notification may be associated with multiple companies.|H302 (100%): Harmful if swallowed [Warning Acute toxicity, oral]|P264, P270, P301+P312, P330, and P501|Aggregated GHS information provided by 43 companies from 1 notifications to the ECHA C&L Inventory.
Drug Information
Kuvan is indicated for the treatment of hyperphenylalaninaemia (HPA) in adults and paediatric patients of all ages with phenylketonuria (PKU) who have been shown to be responsive to such treatment.Kuvan is also indicated for the treatment of hyperphenylalaninaemia (HPA) in adults and paediatric patients of all ages with tetrahydrobiopterin (BH4) deficiency who have been shown to be responsive to such treatment.|Treatment of hyperphenylalaninemia
1-butanone, 1-(2,4,5-trihydroxyphenyl)
Sapropterin dihydrochloride Use and Manufacturing
A natural cofactor for phenylalanine hydroxylase, tyrosine hydroxylase, tryptophan hydroxylase, and nitric oxide synthase. An essential cofactor for the production of neurotransmitters such as catecholamines, serotonin, and nitric oxide. Increasing
Human drugs -> Kuvan -> EMA Drug Category|Other alimentary tract and metabolism products -> Human pharmacotherapeutic group|Human Drugs -> EU pediatric investigation plans|Human Drugs -> FDA Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book) -> Active Ingredients
Computed Properties
Molecular Weight:314.17
Hydrogen Bond Donor Count:8
Hydrogen Bond Acceptor Count:6
Rotatable Bond Count:2
Exact Mass:313.0708448
Monoisotopic Mass:313.0708448
Topological Polar Surface Area:132
Heavy Atom Count:19
Complexity:405
Defined Atom Stereocenter Count:3
Covalently-Bonded Unit Count:3
Compound Is Canonicalized:Yes
Drug Function and Efficacy
Not yet clear
Registered Holders
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MAITHRI DRUGS PRIVATE LTD
Active
United States
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HONOUR LAB LTD
Active
United States
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BIOPHORE INDIA PHARMACEUTICALS PVT LTD
Active
United States
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