References

NCBI Bookshelf. A service of the National Library of Medicine, National Institutes of Health.

Clinical Review Report: Cysteamine delayed-release capsules (Procysbi): Horizon Pharma Ireland Ltd. Indication: For the treatment of nephropathic cystinosis [Internet] Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2018 Feb.

Cover of Clinical Review Report: Cysteamine delayed-release capsules (Procysbi)

Clinical Review Report: Cysteamine delayed-release capsules (Procysbi): Horizon Pharma Ireland Ltd. Indication: For the treatment of nephropathic cystinosis [Internet]

Show details

Contents

< PrevNext >

References

1.: Ariceta G, Camacho JA, Fernandez-Obispo M, Fernandez-Polo A, Gamez J, Garcia-Villoria J, et al. Cystinosis in adult and adolescent patients: recommendations for the comprehensive care of cystinosis. Nefrologia. 2015;35(3):304–21. [PubMed: 26523297]
2.: Cherqui S. Cysteamine therapy: a treatment for cystinosis, not a cure. Kidney Int [Internet]. 2012 Jan [cited 2017 Oct 3];81(2):127–9. Available from: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3386783 [PMC free article: PMC3386783] [PubMed: 22205430]
3.: Elmonem MA, Veys KR, Soliman NA, Van Dyck M, van den Heuvel LP, Levtchenko E. Cystinosis: a review. Orphanet J Rare Dis [Internet]. 2016 Apr 22 [cited 2017 Oct 3];11:47. Available from: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4841061
4.: Wilmer MJ, Schoeber JP, van den Heuvel LP, Levtchenko EN. Cystinosis: practical tools for diagnosis and treatment. Pediatr Nephrol [Internet]. 2011 Feb [cited 2017 Oct 3];26(2):205–15. Available from: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3016220 [PMC free article: PMC3016220] [PubMed: 20734088]
5.: Center for Drug Evaluation and Research, U.S. Food and Drug Administration. Medical review(s). In: Procysbi (cysteamine bitartrate). Company: Raptor Therapeutics, Inc. Application no.: 203389. Approval date: 04/30/2013 [Internet]. Rockville (MD): FDA; 2013 [cited 2017 Aug 25]. (FDA drug approval package). Available from: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000TOC.cfm
6.: Clinical Study Report (Amended): RP103-03. A randomized crossover, pharmacokinetic and pharmacodynamic study to determine the safety and efficacy of cysteamine bitartrate delayed-release capsules (RP103), compared to Cystagon® in subjects with nephropathic cystinosis [CONFIDENTIAL internal manufacturer’s report]. Novato (CA): Raptor Pharmaceuticals Inc.; 2012 Dec 26.
7.: Levtchenko E. Cystinosis: improved medical care and new insights into pathogenesis. The Netherlands: 2005.
8.: Nesterova G, Gahl WA. Cystinosis: the evolution of a treatable disease. Pediatr Nephrol [Internet]. 2013 Jan [cited 2017 Nov 7];28(1):51–9. Available from: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3505515 [PMC free article: PMC3505515] [PubMed: 22903658]
9.: De Braekeleer M. Hereditary disorders in Saguenay-Lac-St-Jean (Quebec, Canada). Hum Hered. 1991;41(3):141–6. [PubMed: 1937486]
10.: Besouw M, Emma F, Levtchenko E. Management of nephropathic cystinosis. Expert Opin Orphan Drugs. 2013;1(2):1031–9.
11.: Morel T, Arickx F, Befrits G, Siviero P, Van Der Meijden C, Xoxi E, et al. Reconciling uncertainty of costs and outcomes with the need for access to orphan medicinal products: A comparative study of managed entry agreements across seven European countries. Orphanet Journal of Rare Diseases [Internet]. 2013 [cited 2017 Sep 26];8(1). Available from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3882782/pdf/1750-1172-8-198.pdf [PMC free article: PMC3882782] [PubMed: 24365263]
12.: Karpman D, Hoglund P. Orphan drug policies and use in pediatric nephrology. Pediatr Nephrol [Internet]. 2017 Jan [cited 2017 Sep 27];32(1):1–6. Available from: https://link.springer.com/content/pdf/10.1007%2Fs00467-016-3520-4.pdf [PubMed: 27738765]
13.: Markello TC, Bernardini IM, Gahl WA. Improved renal function in children with cystinosis treated with cysteamine. N Engl J Med. 1993 Apr 22;328(16):1157–62. [PubMed: 8455682]
14.: Center for Drug Evaluation and Research, U.S. Food and Drug Administration. Statistical review(s). In: Procysbi (cysteamine bitartrate). Company: Raptor Therapeutics, Inc. Application no.: 203389. Approval date: 04/30/2013 [Internet]. Rockville (MD): FDA; 2013 [cited 2017 Aug 25]. (FDA drug approval package). Available from: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2013/203389Orig1s000TOC.cfm
15.: PrProcysbi^™ (cysteamine delayed-release capsules): 25mg and 75mg cysteamine (as cysteamine bitartrate, also called mercaptamine bitartrate) [product monograph]. Dublin (IR): Horizon Pharma Ireland Ltd.; 2017 Jun 8.
16.: Langman CB, Greenbaum LA, Sarwal M, Grimm P, Niaudet P, Deschenes G, 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 [Internet]. 2012 Jul [cited 2017 Oct 3];7(7):1112–20. Available from: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3386675 [PMC free article: PMC3386675] [PubMed: 22554716]
17.: CDR submission: Procysbi (cysteamine), 25 or 75mg delayed-release capsules. Company: Horizon Pharma Ireland Ltd. [CONFIDENTIAL manufacturer’s submission]. Dublin (IR): Horizon Pharma; 2017 Jul.
18.: Erratum: correction: Langman CB, Greenbaum LA, Sarwal M, Grimm P, Niaudet P, DEschenes G, Cornelissen E, Morin D, Cochat P, Matossian D, Gaillard S, Bagger MJ, Rious P: A randomized controlled crossover trial with delayed-release cysteamine bitartrate in nephropathhic cysinosis: effectiveness on white blood cell cystine levels and comparison of safety. Clin J Am Soc Nephrol. 2012;7:1112–1120. Clin J Am Soc Nephrol. 2013 Mar;8:468. [PMC free article: PMC3386675] [PubMed: 22554716]
19.: Besouw M, Tangerman A, Cornelissen E, Rioux P, Levtchenko E. Halitosis in cystinosis patients after administration of immediate-release cysteamine bitartrate compared to delayed-release cysteamine bitartrate. Mol Genet Metab. 2012 Sep;107(1–2):234–6. [PubMed: 22832073]
20.: Besouw MT, Levtchenko EN. Improving the prognosis of nephropathic cystinosis. Int J Nephrol Renovascular Dis [Internet]. 2014 [cited 2017 Oct 3];7:297–302. Available from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4109637/pdf/ijnrd-7-297.pdf [PMC free article: PMC4109637] [PubMed: 25114580]
21.: Dohil R, Cabrera BL. Treatment of cystinosis with delayed-release cysteamine: 6-year follow-up. Pediatr Nephrol. 2013 Mar;28(3):507–10. [PubMed: 23001048]
22.: Brodin-Sartorius A, Tete MJ, Niaudet P, Antignac C, Guest G, Ottolenghi C, et al. Cysteamine therapy delays the progression of nephropathic cystinosis in late adolescents and adults. Kidney Int [Internet]. 2012 Jan [cited 2017 Sep 11];81(2):179–89. Available from: http://www.sciencedirect.com/science/article/pii/S0085253815552678?via%3Dihub [PubMed: 21900880]
23.: Manz F, Gretz N, Broyer M, Geisert J, Grote K, Weber HP, et al. Progression of chronic renal failure in a historical group of patients with nephropathic cystinosis. Pediatric Nephrology. 1994;8(4):466–71. [PubMed: 7947039]
24.: Nesterova G, Williams C, Bernardini I, Gahl WA. Cystinosis: renal glomerular and renal tubular function in relation to compliance with cystine-depleting therapy. Pediatric Nephrology. 2015 Jun;30(6):945–51. [PubMed: 25526929]
25.: Medic G, van der Weijden M, Karabis A, Hemels M. A systematic literature review of cysteamine bitartrate in the treatment of nephropathic cystinosis. Curr Med Res Opin. 2017 Aug 3;1–12. [PubMed: 28692321]
26.: Ahlenstiel-Grunow T, Kanzelmeyer NK, Froede K, Kreuzer M, Drube J, Lerch C, et al. Switching from immediate- to extended-release cysteamine in nephropathic cystinosis patients: a retrospective real-life single-center study. Pediatr Nephrol. 2017 Jan;32(1):91–7. [PubMed: 27350621]
27.: Cysteamine (new preparation). A real advance in the treatment of cystinosis. Prescrire Int. 1999;8(41):67–70. [PubMed: 10558441]
28.: Greco M, Brugnara M, Zaffanello M, Taranta A, Pastore A, Emma F. Long-term outcome of nephropathic cystinosis: a 20-year single-center experience. Pediatr Nephrol. 2010 Dec;25(12):2459–67. [PubMed: 20803298]
29.: Gahl WA, Balog JZ, Kleta R. Nephropathic cystinosis in adults: natural history and effects of oral cysteamine therapy. Ann Intern Med. 2007 Aug 21;147(4):242–50. [PubMed: 17709758]
30.: Cochat P. A cohort of patients with cystinosis: compliance to cysteamine and neurological complications. Descriptive analysis report: compliace at 1 year. Centre d’Investigation Clinique de Lyon; 2016 Apr 15. Lyon (FR). (CrYSTobs Compliance report).
31.: Devereux G, Steele S, Griffiths K, Devlin E, Fraser-Pitt D, Cotton S, et al. An open-label investigation of the pharmacokinetics and tolerability of oral cysteamine in adults with cystic fibrosis. Clin Drug Invest [Internet]. 2016 Aug [cited 2017 Sep 11];36(8):605–12. Available from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4951511/ [PMC free article: PMC4951511] [PubMed: 27153825]
32.: Gahl WA, Reed GF, Thoene JG, Schulman JD, Rizzo WB, Jonas AJ, et al. Cysteamine therapy for children with nephropathic cystinosis. N Engl J Med. 1987 Apr 16;316(16):971–7. [PubMed: 3550461]
33.: Gahl WA, Thoene JG, Schneider JA, O’Regan S, Kaiser-Kupfer MI, Kuwabara T. Cystinosis: progress in a prototypic disease. Ann Intern Med. 1988;109(7):557–69. [PubMed: 3048161]
34.: Tsilou ET, Thompson D, Lindblad AS, Reed GF, Rubin B, Gahl W, 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 [Internet]. 2003 Jan [cited 2017 Sep 11];87(1):28–31. Available from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1771471/ [PMC free article: PMC1771471] [PubMed: 12488257]
35.: Langman CB, Greenbaum LA, Grimm P, Sarwal M, Niaudet P, Deschenes G, et al. Quality of life is improved and kidney function preserved in patients with nephropathic cystinosis treated for 2 years with delayed-release cysteamine bitartrate. J Pediatr [Internet]. 2014 Sep [cited 2017 Sep 26];165(3):528–33. Available from: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4181581 [PMC free article: PMC4181581] [PubMed: 24948347]
36.: Bertholet-Thomas A, Berthiller J, Tasic V, Kassai B, Otukesh H, Greco M, et al. Worldwide view of nephropathic cystinosis: results from a survey from 30 countries. BMC Nephrology [Internet]. 2017 Jul 3 [cited 2017 Sep 11];18(1):210. Available from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5496396/ [PMC free article: PMC5496396] [PubMed: 28673276]
37.: Van Stralen KJ, Emma F, Jager KJ, Verrina E, Schaefer F, Laube GF, et al. Improvement in the renal prognosis in nephropathic cystinosis. Clin J Am Soc Nephrol [Internet]. 2011 Oct [cited 2017 Oct 3];6(10):2485–91. Available from: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3359556 [PMC free article: PMC3359556] [PubMed: 21868618]
38.: Varni JW, Seid M, Rode CA. The PedsQL: measurement model for the pediatric quality of life inventory. Med Care. 1999 Feb;37(2):126–39. [PubMed: 10024117]
39.: Varni JW, Seid M, Kurtin PS. PedsQL 4.0: reliability and validity of the Pediatric Quality of Life Inventory version 4.0 generic core scales in healthy and patient populations. Med Care. 2001 Aug;39(8):800–12. [PubMed: 11468499]
40.: Varni JW, Burwinkle TM, Seid M. The PedsQL as a pediatric patient-reported outcome: reliability and validity of the PedsQL Measurement Model in 25,000 children. Expert Rev Pharmacoecon Outcomes Res. 2005 Dec;5(6):705–19. [PubMed: 19807613]
41.: Varni JW. The PedsQL™: measurement model for the Pediatric Quality of Life Inventory™ [Internet]. Lyon (FR): Mapi Research Trust; 2017. [cited 2017 Oct 10]. Available from: http://www.pedsql.org/index.html
42.: Varni JW, Seid M, Knight TS, Uzark K, Szer IS. The PedsQL 4.0 Generic Core Scales: sensitivity, responsiveness, and impact on clinical decision-making. J Behav Med. 2002 Apr;25(2):175–93. [PubMed: 11977437]
43.: Desai AD, Zhou C, Stanford S, Haaland W, Varni JW, Mangione-Smith RM. Validity and responsiveness of the pediatric quality of life inventory (PedsQL) 4.0 generic core scales in the pediatric inpatient setting. Jama Pediatr [Internet]. 2014 Dec [cited 2017 Oct 3];168(12):1114–21. Available from: http://jamanetwork.com/journals/jamapediatrics/fullarticle/1913622 [PubMed: 25347549]
44.: Ware JE, Jr., Sherbourne CD. The MOS 36-item short-form health survey (SF-36). I. Conceptual framework and item selection. Med Care. 1992 Jun;30(6):473–83. [PubMed: 1593914]
45.: Arends M, Hollak CE, Biegstraaten M. Quality of life in patients with Fabry disease: a systematic review of the literature. Orphanet J Rare Dis [Internet]. 2015 Jun 16 [cited 2017 Jul 24];10:77. Available from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4501376/pdf/13023_2015_Article_296.pdf [PMC free article: PMC4501376] [PubMed: 26076709]
46.: Mease PJ, Menter MA. Quality-of-life issues in psoriasis and psoriatic arthritis: outcome measures and therapies from a dermatological perspective. J Am Acad Dermatol. 2006 Apr;54(4):685–704. [PubMed: 16546593]
47.: Hays RD, Morales LS. The RAND-36 measure of health-related quality of life. Ann Med. 2001 Jul;33(5):350–7. [PubMed: 11491194]
48.: Samsa G, Edelman D, Rothman ML, Williams GR, Lipscomb J, Matchar D. Determining clinically important differences in health status measures: a general approach with illustration to the Health Utilities Index Mark II. PharmacoEconomics. 1999 Feb;15(2):141–55. [PubMed: 10351188]
49.: Maruish ME, editor. User’s manual for the SF-36v2 health survey. 3rd ed. Lincoln (RI): Quality Metric Inc.; 2011. Determining important differences in scores.
50.: Emma F, Nesterova G, Langman C, Labbe A, Cherqui S, Goodyer P, et al. Nephropathic cystinosis: an international consensus document. Nephrol Dial Transplant [Internet]. 2014 Sep [cited 2017 Oct 3];29 Suppl 4:iv87–iv94. Available from: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4158338 [PMC free article: PMC4158338] [PubMed: 25165189]
51.: Gertsman I, Johnson WS, Nishikawa C, Gangoiti JA, Holmes B, Barshop BA. Diagnosis and monitoring of cystinosis using immunomagnetically purified granulocytes. Clin Chem [Internet]. 2016 May [cited 2017 Oct 3];62(5):766–72. Available from: http://clinchem.aaccjnls.org/content/clinchem/62/5/766.full.pdf [PubMed: 26980209]
52.: Gahl WA, Schneider JA, Schulman JD, Thoene JG, Reed GF. Predicted reciprocal serum creatinine at age 10 years as a measure of renal function in children with nephropathic cystinosis treated with oral cysteamine. Pediatr Nephrol [Internet]. 1990 Mar [cited 2017 Oct 10];4(2):129–35. Available from: https://deepblue.lib.umich.edu/bitstream/handle/2027.42/47828/467_2004_Article_BF00858823.pdf?sequence=1&isAllowed=y [PubMed: 2397178]
53.: Bava S, Theilmann RJ, Sach M, May SJ, Frank LR, Hesselink JR, et al. Developmental changes in cerebral white matter microstructure in a disorder of lysosomal storage. Cortex [Internet]. 2010 Feb [cited 2017 Oct 10];46(2):206–16. Available from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3351112/pdf/nihms-176251.pdf [PMC free article: PMC3351112] [PubMed: 19427638]
54.: de Graaf-Hess A, Trijbels F, Blom H. New method for determining cystine in leukocytes and fibroblasts. Clin Chem. 1999 Dec;45(12):2224–8. [PubMed: 10585356]
55.: Levtchenko E, de Graaf-Hess A, Wilmer M, van den Heuvel L, Monnens L, Blom H. Comparison of cystine determination in mixed leukocytes vs polymorphonuclear leukocytes for diagnosis of cystinosis and monitoring of cysteamine therapy. Clin Chem. 2004 Sep;50(9):1686–8. [PubMed: 15331510]
56.: Clinical Study Report: RP103-04. A long-term, open-label, safety and efficacy study of cysteamine bitartrate delayed-release capsules (rp103) in patients with cystinosis. Interim CSR [CONFIDENTIAL internal manufacturer’s report]. Novato (CA): Raptor Pharmaceuticals Inc.; 2015 Nov 11.
57.: Preliminary Study Report: RP103-07. A long-term, open-label, safety, tolerability and superior effectiveness study of cysteamine bitartrate delayed-release capsules (RP103) in patients with cystinosis [CONFIDENTIAL internal manufacturer’s report]. Novato (CA): Raptor Pharmaceuticals Inc.; 2015 Oct 16.

The copyright and other intellectual property rights in this document are owned by CADTH and its licensors. These rights are protected by the Canadian Copyright Act and other national and international laws and agreements. Users are permitted to make copies of this document for non-commercial purposes only, provided it is not modified when reproduced and appropriate credit is given to CADTH and its licensors.

Except where otherwise noted, this work is distributed under the terms of a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International licence (CC BY-NC-ND), a copy of which is available at http://creativecommons.org/licenses/by-nc-nd/4.0/

Bookshelf ID: NBK533999

Contents

< PrevNext >

PubReader
Print View
Cite this Page
Clinical Review Report: Cysteamine delayed-release capsules (Procysbi): Horizon Pharma Ireland Ltd. Indication: For the treatment of nephropathic cystinosis [Internet] Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2018 Feb. References.
PDF version of this title (1.6M)

Other titles in this collection

CADTH Common Drug Reviews

Related information

PMC
PubMed Central citations
PubMed
Links to PubMed

Recent Activity

Clear Turn Off Turn On

References - Clinical Review Report: Cysteamine delayed-release capsules (Procys...
References - Clinical Review Report: Cysteamine delayed-release capsules (Procysbi)
REFERENCES - Glycerol Phenylbutyrate (Ravicti)
REFERENCES - Glycerol Phenylbutyrate (Ravicti)
Review of the Pharmacoeconomic Submission - Pharmacoeconomic Review Report: dolu...
Review of the Pharmacoeconomic Submission - Pharmacoeconomic Review Report: dolutegravir (Tivicay)
ABBREVIATIONS - Ustekinumab (Stelara)
ABBREVIATIONS - Ustekinumab (Stelara)
Conclusions - Clinical and Economic Review Report: Vedolizumab (ENTYVIO SC)
Conclusions - Clinical and Economic Review Report: Vedolizumab (ENTYVIO SC)

Your browsing activity is empty.

Activity recording is turned off.

Turn recording back on

Bookshelf

Clinical Review Report: Cysteamine delayed-release capsules (Procysbi): Horizon Pharma Ireland Ltd. Indication: For the treatment of nephropathic cystinosis [Internet]

References

Views

Other titles in this collection

Related information

Recent Activity