Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • Research Highlight
  • Published:

Therapy

An extended-release form of cysteamine bitartrate for cystinosis

Nature Reviews Nephrology volume 8, page 374 (2012)Cite this article

Subjects

Treatment of cystinosis necessitates that the cysteamine bitartrate Cystagon® (Mylan Pharmaceuticals, Morgantown, WV, USA) is taken every 6 h. New research, however, has shown that a novel, delayed-release formulation of cysteamine bitartrate, called RP103, which can be taken every 12 h, is noninferior to Cystagon® for maintaining cystine at levels associated with optimal outcomes for patients with this disease.

To compare the efficacy of RP103 with Cystagon®, Langman and colleagues performed an open-label, randomized, controlled, crossover study, in which they randomly assigned 43 patients to receive either RP103 or Cystagon® for 3 weeks. The primary end point was based on the comparison of peak reduced cystine (½ cystine) levels in peripheral white blood cells (WBCs). “We demonstrated that when used on a 12 h basis, RP103 was not inferior to Cystagon® for the ability to keep WBC ½ cystine levels equivalently low”, notes Langman. “Importantly, we did this at a total daily dose of the active agent, cysteamine bitartrate, with RP103 that was only 82% of the total daily dose of Cystagon®.”

This is a preview of subscription content, access via your institution

Access options

Buy this article

  • Purchase on SpringerLink
  • Instant access to the full article PDF.

USD 39.95

Prices may be subject to local taxes which are calculated during checkout

ORIGINAL RESEARCH PAPER

  1. Langman, C. B. et al. A randomized controlled crossover trial with delayed-release cysteamine bitartrate in nephropathic cystinosis: effectiveness on white blood cell cystine levels and comparison of safety. Clin. J. Am. Soc. Nephrol. doi:10.2215/CJN.12321211

Download references

Authors
  1. Susan J. Allison
    View author publications

    Search author on:PubMed Google Scholar

Rights and permissions

Reprints and permissions

About this article

Cite this article

Allison, S. An extended-release form of cysteamine bitartrate for cystinosis. Nat Rev Nephrol 8, 374 (2012). https://doi.org/10.1038/nrneph.2012.105

Download citation

  • Published:

  • Issue date:

  • DOI: https://doi.org/10.1038/nrneph.2012.105

Search

Advanced search

Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing