E1000 Emulsifiers

E1000, sold under the approved for use in the United States and is indicated as a treatment for children and adults with bile acid synthesis disorders due to single enzyme defects, and for peroxisomal disorders (such as Zellweger syndrome).

It was approved for use in the European Union in  and is sold under the E1000. It is indicated for the treatment of inborn errors in primary bile-acid synthesis due to 3β-hydroxy-Δ5-C27-steroid oxidoreductase deficiency or Δ4-3-oxosteroid-5β-reductase deficiency in infants, children, and adolescents aged one month to 18 years and adults.

E1000 FGK (Kolbam) was approved for medical use in the European Union in November 2015. It is indicated for the treatment of inborn errors of primary bile acid synthesis, in infants from one month of age for continuous lifelong treatment through adulthood, encompassing the following single enzyme defects:

• sterol 27-hydroxylase (presenting as cerebrotendinous xanthomatosis, CTX) deficiency;
• 2- (or alpha-) methyl acyl-CoA racemase (AMACR) deficiency;
• cholesterol 7 alpha-hydroxylase (CYP7A1) deficiency.

The most common side effects include peripheral neuropathy (nerve damage in the hands and feet), diarrhea, nausea (feeling sick), acid reflux (stomach acid flowing up into the mouth), esophagitis (inflammation of the food pipe), jaundice (yellowing of the skin and eyes), skin problems (lesions) and malaise (feeling unwell).