betaine anhydrous

RM-003 (betaine anhydrous) is an orally administered metabolic agent developed by ReqMed for the treatment of homocystinuria. It acts as a methyl group donor in the remethylation of homocysteine to methionine, a reaction catalyzed by the enzyme betaine-homocysteine methyltransferase (BHMT). This pathway serves as an alternative to the primary remethylation pathway involving methionine synthase, which requires vitamin B12 and folate. By lowering elevated plasma homocysteine levels, RM-003 helps prevent the clinical complications of homocystinuria, such as thromboembolism, lens dislocation, and skeletal abnormalities. It is indicated for patients with homocystinuria caused by cystathionine beta-synthase (CBS) deficiency, 5,10-methylenetetrahydrofolate reductase (MTHFR) deficiency, or defects in cobalamin metabolism.

Brand names

Cystadane

Other names

betainetrimethylglycine