Suppository formulations as a potential treatment for nephropathic cystinosis

J Pharm Sci. 2012 Oct;101(10):3729-38. doi: 10.1002/jps.23246. Epub 2012 Jul 6.

Abstract

Nephropathic cystinosis is a rare autosomal recessive disease characterised by raised lysosomal levels of cystine in the cells of all the organs. It is treated by the 6-h oral administration of the aminothiol, cysteamine, which has an offensive taste and smell. In an attempt to reduce this frequency and improve the treatment, cysteamine-containing polyethylene glycol suppositories were prepared and evaluated for dissolution and stability. The results demonstrated that cysteamine release was complete after 30 min, and that there was a uniform drug distribution within the formulations. Twelve-month stability tests highlighted a potential incompatibility among some excipients, although stability was demonstrated for the cysteamine suppositories up to 6 months. These suppositories may provide a useful alternative to the current oral therapy for cystinosis.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Chemistry, Pharmaceutical / methods
  • Cysteamine / administration & dosage
  • Cysteamine / chemistry*
  • Cystine / metabolism
  • Cystinosis / drug therapy*
  • Cystinosis / metabolism
  • Drug Stability
  • Excipients / administration & dosage
  • Excipients / chemistry
  • Fanconi Syndrome / drug therapy*
  • Fanconi Syndrome / metabolism
  • Nephrotic Syndrome / drug therapy*
  • Nephrotic Syndrome / metabolism
  • Polyethylene Glycols / administration & dosage
  • Polyethylene Glycols / chemistry
  • Solubility
  • Suppositories / administration & dosage
  • Suppositories / chemistry*

Substances

  • Excipients
  • Suppositories
  • Polyethylene Glycols
  • Cystine
  • Cysteamine