Skip to main content
Log in

Cystadrops® (cysteamine hydrochloride 0.55% viscous eye-drops solution) in treating corneal cystine crystal deposits in patients with cystinosis: a profile of its use

  • Adis Drug Q&A
  • Published:
Drugs & Therapy Perspectives Aims and scope Submit manuscript

An Erratum to this article was published on 20 April 2017

Abstract

Cystadrops® (viscous cysteamine hydrochloride 0.55% eye-drops solution) is the first orphan drug approved in the EU for the treatment of corneal crystal deposits in adults and children aged >2 years with cystinosis. The solution reduced the accumulation of cystine crystals in the cornea and the severity of photophobia in patients of all ages with cystinosis in clinical studies. Instillation adverse effects (e.g. burning and stinging) may occur, but generally are very transient and mild to moderate in severity. Relative to extemporaneously prepared formulations of cysteamine hydrochloride eye drops, Cystadrops® requires less frequent administration (relieving treatment burden), displays better stability (making storage, distribution and use by patients less difficult) and provides reliable standardization and availability.

This is a preview of subscription content, log in via an institution to check access.

Access this article

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Similar content being viewed by others

References

  1. Gahl WA, Kuehl EM, Iwata F, et al. Corneal crystals in nephropathic cystinosis: natural history and treatment with cysteamine eyedrops. Mol Genet Metab. 2000;71(1):100–20.

    Article  CAS  PubMed  Google Scholar 

  2. Radojkovic B. Cysteamine eye drops in the treatment of cystinosis: an Australian perspective. J Pharm Pract Res. 2015;45(4):440–5.

    Article  Google Scholar 

  3. Hohenfellner K. Cystinosis: prevalence, burden, complications, diagnosis, prognosis and management [2016 Ophthalmology Cystinosis Forum: morning session]. EMJ Nephrol. 2017;Suppl X:3–4.

  4. Shams F, Livingstone I, Oladiwura D, et al. Treatment of corneal cystine crystal accumulation in patients with cystinosis. Clin Ophthalmol. 2014;8:2077–84.

    Article  PubMed  PubMed Central  Google Scholar 

  5. Cystagon 50 mg hard capsules: summary of product characteristics. London: European Medicines Agency; 2014.

  6. Procysbi 25 and 75 mg gastro-resistant hard capsules: summary of product characteristics. London: European Medicines Agency; 2016.

  7. Tsygin A. Outlining the benefits of long-term treatment with cysteamine: sharing data [2016 Ophthalmology Cystinosis Forum: morning session]. EMJ Nephrol. 2017;Suppl X:4–5.

  8. Gahl WA, Tietze F, Butler JD, et al. Cysteamine depletes lysosomal cystine by the mechanism of disulfide interchange. Biochem J. 1985;228(3):545–50.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  9. Pisoni RL, Thoene JG, Christensen HN. Detection and characterization of carrier-mediated cationic amino acid transport in lysosomes of normal and cystinotic human fibroblasts. Role in therapeutic cystine removal? J Biol Chem. 1985;260(8):4791–8.

    CAS  PubMed  Google Scholar 

  10. Gahl WA. Early oral cysteamine therapy for nephropathic cystinosis. Eur J Pediatr. 2003;162(Suppl 1):S38–41.

    Article  CAS  PubMed  Google Scholar 

  11. Kleta R, Gahl WA. Pharmacologic treatment of nephropathic cystinosis with cysteamine. Expert Opin Pharmacother. 2004;5(11):2255–62.

    Article  CAS  PubMed  Google Scholar 

  12. Kleta R, Bernardini I, Ueda M, et al. Long term follow-up of well treated nephropathic cystinosis patients. J Pediatr. 2004;145(4):555–60.

    Article  PubMed  Google Scholar 

  13. Nestorova G, Gahl WA. Cystinosis: the evolution of a treatable disease. Pediatr Nephrol. 2013;28:51–9.

    Article  Google Scholar 

  14. Kaiser-Kupfer MI, Gazzo MA, Datiles MB, et al. A randomized placebo-controlled trial of cysteamine eye drops in nephropathic cystinosis. Arch Ophthalmol. 1990;108(5):689–93.

    Article  CAS  PubMed  Google Scholar 

  15. Kaiser-Kupfer MI, Fujikawa L, Kuwabara T, et al. Removal of corneal crystals by topical cysteamine in nephropathic cystinosis. N Engl J Med. 1987;316(13):775–9.

    Article  CAS  PubMed  Google Scholar 

  16. Tsilou E, Thompson D, Lindblad A, et al. A multicentre randomised double masked clinical trial of a new formulation of topical cysteamine for the treatment of corneal cystine crystals in cystinosis. Br J Ophthalmol. 2003;87(1):28–31.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  17. Bradbury JA, Danjoux JP, Voller J, et al. A randomised placebo-controlled trial of topical cysteamine therapy in patients with nephropathic cystinosis. Eye (Lond). 1991;5(Pt 6):755–60.

    Article  Google Scholar 

  18. Iwata F, Kuehl EM, Reed GF, et al. A randomized clinical trial of topical cysteamine disulfide (cystamine) versus free thiol (cysteamine) in the treatment of corneal cystine crystals in cystinosis. Mol Genet Metab. 1998;64(4):237–42.

    Article  CAS  PubMed  Google Scholar 

  19. Soliman NA, El-Baroudy R, Rizk A, et al. Nephropathic cystinosis in children: an overlooked disease. Saudi J Kidney Dis Transpl. 2009;20(3):436–42.

    PubMed  Google Scholar 

  20. Marcano DC, Shin CS, Lee B, et al. Synergistic cysteamine delivery nanowafer as an efficacious treatment modality for corneal cystinosis. Mol Pharm. 2016;13(10):3468–77.

    Article  CAS  PubMed  Google Scholar 

  21. Al-Hemidan A, Shoughy SS, Kozak I, et al. Efficacy of topical cysteamine in nephropathic cystinosis. Br J Ophthalmol. 2017. doi:10.1136/bjophthalmol-2016-309278.

    PubMed  Google Scholar 

  22. Cystaran™ (cysteamine ophthalmic solution) 0.44% sterile: US prescribing information. Gaithersburg (MD): Sigma-Tau Pharmaceuticals, Inc.; 2012.

  23. Recordati announces marketing approval for Cystadrops® [news release]. Milan: Recordati S.p.A; 2017.

  24. Cystadrops (mercaptamine) 3.8 mg/mL eye drops solution: summary of product characteristics. London: European Medicines Agency; 2017.

  25. Simpson JL, Nien CJ, Flynn KJ, et al. Evaluation of topical cysteamine therapy in the CTNS −/− knockout mouse using in vivo confocal microscopy. Mol Vis. 2011;17:2649–54.

    CAS  PubMed  PubMed Central  Google Scholar 

  26. Labbé A, Baudouin C, Deschênes G, et al. A new gel formulation of topical cysteamine for the treatment of corneal cystine crystals in cystinosis: the Cystadrops OCT-1 study. Mol Genet Metab. 2014;111(3):314–20.

    Article  PubMed  Google Scholar 

  27. Labbé A, Baudouin C, Lyang H, et al. Cysteamine hydrochloride for nephropathic cystinosis: open-label phase III study [abstract no. P-323]. Pediatr Nephrol. 2015;30(Suppl):127. doi:10.1007/s00467-015-3158-7.

  28. Liang H. Ophthalmology latest data with gel-like cysteamine eye drops [2016 Ophthalmology Cystinosis Forum: morning session]. EMJ Nephrol. 2017;Suppl X:3–4.

  29. Liang H, Labbé A, Le Mouhaër J, et al. A new viscous cysteamine eye drops treatment for the ophthalmic cystinosis: an open-label randomized comparative phase III pivotal study. Invest Ophthalmol Vis Sci. 2017 (In press).

  30. Sampaolesi R, Zarate J, Sampaolesi J. The glaucomas. Volume 1: pediatric glaucomas. Berlin: Springer; 2009.

  31. Leal I. Ocular cystinosis: clinical experience in adult patients [2016 Ophthalmology Cystinosis Forum: afternoon session]. EMJ Nephrol. 2017;Suppl X:11–2.

  32. Adverse effects in the eye may be caused by a wide range of drugs used to treat ocular and systemic disorders. Drugs Ther Perspect. 2008;24(10):24–7.

  33. American Academy of Ophthalmology. Focal points—excerpt: general principles. 2016. http://www.aao.org. Accessed 17 Mar 2017.

Download references

Acknowledgements

The manuscript was reviewed by: S. Biswas, Manchester Royal Eye Hospital, Manchester, UK; G. Deschênes, Department of Pediatric Nephrology, Robert Debré Hospital, Université Paris Sorbonne Cité, Paris, France; G. Iarrossi, Department of Ophthalmology, Bambino Gesú Children’s Hospital, Rome, Italy; I. Leal, Centro Estudos Ciencias da Visao, Faculty of Medicine, Universidade de Lisboa, Lisbon, Portugal; B. McKenzie, School of Pharmacy and Life Sciences, Robert Gordon University, Aberdeen, UK; B. Radojkovic, Pharmacy Department, Royal Prince Alfred Hospital, Camperdown, Australia. During the peer review process, the manufacturer of Cystadrops® was offered an opportunity to review this article. Changes resulting from comments received were made on the basis of scientific and editorial merit.

Author information

Authors and Affiliations

Authors

Corresponding author

Correspondence to Katherine A. Lyseng-Williamson.

Ethics declarations

Funding

The preparation of this review was not supported by any external funding.

Conflict of interest

K.A. Lyseng-Williamson is a salaried employee of Adis/Springer, is responsible for the article content and declares no conflicts of interest.

Additional information

An erratum to this article is available at http://dx.doi.org/10.1007/s40267-017-0402-1.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Lyseng-Williamson, K.A. Cystadrops® (cysteamine hydrochloride 0.55% viscous eye-drops solution) in treating corneal cystine crystal deposits in patients with cystinosis: a profile of its use. Drugs Ther Perspect 33, 195–201 (2017). https://doi.org/10.1007/s40267-017-0398-6

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1007/s40267-017-0398-6

Keywords

Navigation