Tìm theo
Carglumic Acid
Các tên gọi khác (13 ) :
  • (2S)-2-(Carbamoylamino)pentanedioic acid
  • (S)-2-ureidopentanedioic acid
  • Acide carglumique
  • Acido carglumico
  • Acidum carglumicum
  • Carbaglu
  • Carbamino-L-glutamic acid
  • Carbamylglutamic acid
  • L-N-Carbamoylglutamic acid
  • N-Carbamoyl-L-Glutamic Acid
  • N-Carbamyl-L-glutamate
  • N-Carbamylglutamate
  • Ureidoglutaric acid
Thuốc Gốc
Small Molecule
CAS: 1188-38-1
ATC: A16AA05
CTHH: C6H10N2O5
PTK: 190.154
Carglumic acid is an orphan drug used for the treatment of hyperammonaemia in patients with N-acetylglutamate synthase deficiency. This rare genetic disorder results in elevated blood levels of ammonia, which can eventually cross the blood–brain barrier and cause neurologic problems, cerebral edema, coma, and death. Carglumic acid was approved by the U.S. Food and Drug Administration (FDA) on 18 March 2010.
Nhận Dạng Quốc Tế & Đặc Tính Hóa Học
Công thức hóa học
C6H10N2O5
Phân tử khối
190.154
Monoisotopic mass
190.05897144
InChI
InChI=1S/C6H10N2O5/c7-6(13)8-3(5(11)12)1-2-4(9)10/h3H,1-2H2,(H,9,10)(H,11,12)(H3,7,8,13)/t3-/m0/s1
InChI Key
InChIKey=LCQLHJZYVOQKHU-VKHMYHEASA-N
IUPAC Name
(2S)-2-(carbamoylamino)pentanedioic acid
Traditional IUPAC Name
carglumic acid
SMILES
NC(=O)N[C@@H](CCC(O)=O)C(O)=O
Độ hòa tan
1.91e+01 g/l
logP
-1.097
logS
-1
pKa (strongest acidic)
3.36
pKa (Strongest Basic)
-2.3
PSA
129.72 Å2
Refractivity
39.41 m3·mol-1
Polarizability
16.78 Å3
Rotatable Bond Count
5
H Bond Acceptor Count
5
H Bond Donor Count
4
Physiological Charge
-2
Number of Rings
0
Bioavailability
1
Rule of Five
true
Dược Lực Học : The median Tmax of Carbaglu was 3 hours (range: 2-4). The initial daily dose ranges from 100 to 250 mg/kg, adjusted thereafter to maintain normal plasma levels of ammonia.
Cơ Chế Tác Dụng : Carglumic acid is an orphan drug used for the treatment of hyperammonaemia in patients with N-acetylglutamate synthase deficiency. This rare genetic disorder results in elevated blood levels of ammonia, which can eventually cross the blood–brain barrier and cause neurologic problems, cerebral edema, coma, and death. Carglumic acid was approved by the U.S. Food and Drug Administration (FDA) on 18 March 2010. Carglumic acid is a synthetic structural analogue of N-acetylglutamate (NAG), which is an essential allosteric activator of the liver enzyme carbamoyl phosphate synthetase 1 (CPS1). CPS1 is found in the mitochondria and is the first enzyme of the urea cycle, which converts ammonia into urea. Carglumic acid acts as a replacement for NAG in NAGS deficiency patients by activating CPS1 but it does not help to regulate the urea cycle.
Dược Động Học :
▧ Absorption :
30% bioavailability; Cmax, mean, 100 mg/kg dose = 2.6 μg/mL (range of 1.9 - 4.8) Carglumic acid is not subject to to intracellular degradation.
▧ Volume of Distribution :
The apparent volume of distribution was 2657 L (range: 1616-5797).
▧ Metabolism :
A proportion of carglumic acid may be metabolized by the intestinal bacterial flora. The likely end product of carglumic acid metabolism is carbon dioxide, eliminated through the lungs.
▧ Route of Elimination :
Following administration of a single radiolabeled oral dose of 100 mg/kg of body weight, 9% of the dose was excreted unchanged in the urine and up to 60% of the dose was excreted unchanged in the feces.
▧ Half Life :
Median values for the terminal half-life was 5.6 hours (range 4.3-9.5).
▧ Clearance :
The apparent total clearance was 5.7 L/min (range 3.0-9.7), the renal clearance was 290 mL/min (range 204-445), and the 24-hour urinary excretion was 4.5 % of the dose (range 3.5-7.5).
Độc Tính : LD50, oral, mouse: >1000 mg/kg
Chỉ Định : For the treatment of acute and chronic hyperammonaemia in patients with N-acetylglutamate synthase (NAGS) deficiency. This enzyme is an important component of the urea cycle to prevent build up of neurotoxic ammonium in the blood.
Liều Lượng & Cách Dùng : Tablet, for suspension - Oral - 200 mg
Dữ Kiện Thương Mại
Nhà Sản Xuất
  • Công ty : Orphan Europe
    Sản phẩm biệt dược : Carbaglu
Tài Liệu Tham Khảo Thêm
drugbank
http://www.drugbank.ca/drugs/DB06775
PubChem Compound
http://pubchem.ncbi.nlm.nih.gov/summary/summary.cgi?sid=121396
PubChem Substance
http://pubchem.ncbi.nlm.nih.gov/summary/summary.cgi?sid=99443290
KEGG Compound
http://www.genome.jp/dbget-bin/www_bget?cpd:C05829
KEGG Drug
http://www.genome.jp/dbget-bin/www_bget?drug:D07130
ChemSpider
http://www.chemspider.com/Chemical-Structure.108351.html
National Drug Code Directory
http://www.hipaaspace.com/Medical_Billing/Coding/National.Drug.Codes/PDF/52276-312-05
PharmGKB
http://www.pharmgkb.org/drug/PA165958402
Wikipedia
http://en.wikipedia.org/wiki/Carglumic_acid
RxList
http://www.rxlist.com/carbaglu-drug.htm
Drugs.com
http://www.drugs.com/carbaglu.html
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