Carglumic acid
From The Cure For The Needy
Contents |
Carglumic Acid (Carbaglu)
Effective Against
Hyperammonaemia in patients with N-acetylglutamate synthase deficiency
Cost
"The cost of treatment relates to the patient’s body weight, so ranges from approximately £40K for a 1 year old child rising to £245K for a 60kg adult per annum."
Information from http://www.nelm.nhs.uk/en/NeLM-Area/Evidence/Drug-Specific-Reviews/495911/
Based on exchange rates as of June 26, 2009, £40K = 66K USD and £245K = 404K USD.
Reported Routes of Synthesis
Reaction of L-glutamic acid (I) with KOCN yields the title compound (1).
Carbaglu - European Public Assessment Report (EPAR)
Web Site of The European Agency for the Evaluation of Medicinal Products (EMEA) 2003, January 24
"Carglumic acid is synthesised by reaction of L-glutamic acid and potassium cyanate in an aqueous
media in a single step, followed by neutralisation with hydrochloric acid and purification by
recrystallisations in water. No organic solvents or metal catalysts are used during the synthetic
procedure.
The potential impurities originating from the route of synthesis as well as from the degradation of the active substance are hydantoin-5-propionic acid and diaza-1,3-dione-2,4-carboxy-7-cycloheptane. The optical isomer of the active substance, N-carbamoyl-D-glutamic acid might also be present. No residuals of the starting materials, L- glutamic acid and potassium cyanate, were detected in carglumic acid. Residual levels of cyanide ions possibly originating from potassium cyanate were also tested and not detected. Other unidentified substances appear only under stress conditions to which the active substance or the finished product is never subject."
European Public Assessment Report on Carbaglu, Scientific Discussion, accessed 6/30/2009
If PDF doesn't show, try the following website http://www.emea.europa.eu/humandocs/Humans/EPAR/carbaglu/carbaglu.htm
Cost of Reagents:
L-glutamic acid from Sigma-Aldrich: 13 USD (2.5 g)
Potassium cyanate from Sigma-Aldrich: 25.2 USD (5g)