Clinical evaluation of natalizumab for formulary consideration

Expert Opinion on Biological Therapy

Volumn 10, Issue 8, 2010, Pages 1279-1287

  Bivins, Ashley ^a   Hou, Karolyn ^a   Ayesu, Nana ^a   Ellsworth Jr , Brian ^a   Montenegro, Susan ^a   Tu, Xiao ^a   Boyle, Cynthia ^a   Dowling, Thomas ^a   Shaya, Fadia T ^a  

^a UNIVERSITY OF MARYLAND SCHOOL OF PHARMACY   (United States)

Author keywords

Comparative effectiveness; Crohn's disease; Multiple sclerosis; Tysabri

Indexed keywords

BETA1A INTERFERON; GADOLINIUM; NATALIZUMAB; PLACEBO; VERY LATE ACTIVATION ANTIGEN 4; IMMUNOSUPPRESSIVE AGENT; MONOCLONAL ANTIBODY;

ANXIETY; CLINICAL EVALUATION; CLINICAL TRIAL; CROHN DISEASE; DRUG EFFICACY; DRUG HYPERSENSITIVITY; DRUG MECHANISM; DRUG SAFETY; HUMAN; INFECTION; MONOTHERAPY; MULTIPLE SCLEROSIS; NUCLEAR MAGNETIC RESONANCE IMAGING; PATIENT MONITORING; PERIPHERAL EDEMA; PHARYNGITIS; PROGRESSIVE MULTIFOCAL LEUKOENCEPHALOPATHY; REMISSION; REVIEW; SIDE EFFECT; SINUS CONGESTION; TREATMENT OUTCOME; TREATMENT RESPONSE; CLINICAL TRIAL (TOPIC); EVIDENCE BASED MEDICINE; MULTIPLE SCLEROSIS, RELAPSING-REMITTING; PUBLICATION;

ANTIBODIES, MONOCLONAL; ANTIBODIES, MONOCLONAL, HUMANIZED; CLINICAL TRIALS AS TOPIC; CROHN DISEASE; EVIDENCE-BASED MEDICINE; FORMULARIES AS TOPIC; HUMANS; IMMUNOSUPPRESSIVE AGENTS; MULTIPLE SCLEROSIS, RELAPSING-REMITTING; TREATMENT OUTCOME;

EID: 77954677990     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712598.2010.505234     Document Type: Review

References (73)

1. Knigge KL. Inflammatory bowel disease. Clin Cornerstone 2002;4:49-60
2. Loftus EV Jr. Clinical epidemiology of inflammatory bowel disease: incidence, prevalence, and environmental influences. Gastroenterology 2004;126:1504-1517
3. Karlinger K, Gyorke T, Mako E, et al. The epidemiology and the pathogenesis of inflammatory bowel disease. Eur J Radiol 2000;35:154-167
4. Carter MJ, Lobo AJ, Travis SP. Guidelines for the management of inflammatory bowel disease in adults. Gut 2004;53(suppl 5):V1-16
5. Hanauer SB. Turning traditional treatment strategies on their heads: current evidence for "stepup" versus "top-down". Rev Gastroenterol Disord 2007;7(suppl 2):s17-22
6. Hommes D, Baert F, van Assche G, et al. A randomized controlled trial evaluating the ideal medical management for Crohn's Disease (CD): top-down versus step-up strategies. Gastroenterology 2005;128(suppl 2):A-577
7. Multiple Sclerosis International Federation. MS - the disease. Quick facts. Available from: http://www.msif. org/en/about-ms/index.html.
8. What is multiple sclerosis. National Multiple Sclerosis Society. New York. Available from: http://www. nationalmssociety.org/about-multiple- sclerosis/what-we-know-about-ms/what-is-ms/index.aspx [Last accessed 29 June 2010]
9. Leary SM, Porter B, Thompson AJ. Multiple sclerosis: diagnosis and the management of acute relapse. Postgrad Med J 2005;81:302-308
10. Compston A, Ebers G, Lassman H, et al. McAlpine's multiple sclerosis. 3rd edition. Churchill Livingstone, London, UK; 1988
11. Compston A. Coles A. Multiple sclerosis. Lancet 2002;350:1221-1231
12. Noseworthy JH, Lucchinetti C, Rodriguez M, et al. Multiple sclerosis. N Engl J Med 2000;343:938-952
13. Boster A, Gilles E, Adil J, et al. Intense immunosupression in patients with rapidly worsening multiple sclerosis: treatment guidelines for the clinician. Lancet Neurol 2008;7:173-183
14. National Collaborating Centre for Chronic Conditions. Multiple sclerosis. National clinical guideline for diagnosis and management in primary and secondary care. National Institute for Clinical Excellence (NICE), London. UK; 2004
15. Elovaara I, Apostolsk S, van doorn P, et al. EFNS guidelines for the use of intravenous immunoglobulin in treatment of neurological diseases. Eur J Neurol 2008;15:893-908
16. Sellebjerg F, Barnes D, Fillippini G, et al. EFNS guideline on treatment of multiple sclerosis relapses: report of an EFNS task force on treatment of multiple sclerosis relapses. Eur J Neurol 2005;12:939-946
17. Assessment: the use of natalizumab (Tysabri) for the treatment of multiple sclerosis (an evidence-based review). Report of the Therapeutics and Technology Assessment Subcommittee of the American Academy of Neurology. National Guideline Clearinghouse; Rockville, MD; 2010. Available from: http://www.guideline.gov/summary/ summary.aspx?doc-id=13011&nbr=6705&ss= 6&xl=999. [Last accessed 29 June 2010]
18. Vajkoczy P, Laschinger M, Engelhardt B. Alpha4-integrin-VCAM-1 binding mediates G protein-independent capture of encephalitogenic T cell blasts to CNS white matter microvessels. J Clin Invest 2001;108:557-565
19. Massart C, Gibassier J, Oger J, et al. Neutralizing antibodies to interferon beta in multiple sclerosis: analytical evaluation for validation of a cytopathtic effect assay. Clinica Chimica Acta 2007;377:185-191
20. European Public Assessment Report (EPAR) Tysabri. European Medicines Agency, London, UK; 27 June 2006. Available from: http://www.ema. europa.eu/humandocs/PDFs/ EPAR/tysabri/H-603-en1.pdf. [Last accessed 29 June 2010]
21. Tysabri (natalizumab) Dossier Crohn's disease, Biogen Idec, Inc. and Elan Pharmaceuticals, Inc., 2008
22. Tysabri (natalizumab) Dossier Multiple Sclerosis, Biogen Idec, Inc. and Elan Pharmaceuticals, Inc., 2008
23. Tysabri Prescribing Information. Biogen Idec, Inc., Cambridge, MA; 2006. Available from: http://www.biogenidec.com/site/ TYSABRI-PI-MedGuide.pdf. [Last accessed 29 June 2010]
24. Minagar A, Alexander JS. Blood-brain barrier disruption in multiple sclerosis. Mult Scler 2003;9:540-549
25. Carlos TM, Harlan JM. Leukocyte-endothelial adhesion molecules. Blood 1994;84:2068-2101
26. von Andrian UH, Engelhardt B. Alpha4 integrins as therapeutic targets in autoimmune disease. N Engl J Med 2003;348:68-72
27. Frenette PS, Wagner DD. Adhesion molecules: part I. N Engl J Med 1996;334:1526-1529
28. Frenette PS, Wagner DD. Adhesion molecules: part II: blood vessels and blood cells. N Engl J Med 1996;335:43-45
29. Hickey WF, Hsu BL, Kimura H. T-lymphocyte entry into the central nervous system. J Neurosci Res 1991;28:254-260
30. Vajkoczy P, Laschinger M, Engelhardt B. Alpha4-integrin-VCAM-1 binding mediates G protein-independent capture of encephalitogenic T cell blasts to CNS white matter microvessels. J Clin Invest 2001;108:557-565
31. Rudick RA, Sandrock A. Natalizumab: alpha4-integrin antagonist selective adhesion molecule inhibitors for MS. Expert Rev Neurother 2004;4(4):571-580
32. Brudek T, Christensen T, Aagaard L, et al. B cells and monocytes from patients with active multiple sclerosis exhibit increased surface expression of both HERV-H Env and HERV-W Env, accompanied by increased seroreactivity. Retrovirology 2009;6:104
33. Sheremata WA, Minagar A, Alexanaer JS, et al. The role of alpha-4 integrin in the etiology of multiple sclerosis: Current knowledge and therapeutic implications. CNS Drugs 2005;19:909-922
34. Linda H, von Heijne A, Major EO, et al. Progressive multifocal lekoencephalopathy after natalizumab monotherapy. N Engl J Med 2009;361:1081-1087
35. The Tysabri TOUCH® Prescribing Program. Biogen Idec, Weston, MA; Available from: http://www.tysabri.com/ tysbProject/tysb.portal/-baseurl/ threeColLayout/SCSRepository/en-US/ tysb/home/treatment-with-tysabri/ touch-prescribing-program.xml. [Last accessed 29 June 2009]
36. Annex: Conditions or restrictions with regard to the safe and effective use of the medicinal product to be implemented by the member states. European Medicines Agency, London, UK; [22 December 2006]. Available at: http://www.ema.europa.eu/humandocs/ PDFs/EPAR/tysabri/H-603-Annex-en.pdf
37. Polman CH, O'Connor PW, Havrdova E, et al. A randomized, placebo-controlled trial of natalizumab for relapsing multiple sclerosis. N Engl J Med 2006;354:899-910
38. Rudick RA, Stuart WH, Calabresi PA, et al. Natalizumab plus interferon beta-1a for relapsing multiple sclerosis. N Engl J Med 2006;354:911-923
39. Targan SR, Feagan BG, Fedorak RN, et al. International Efficacy of Natalizumab in Crohn's Disease Response and Remission (ENCORE) Trial Group. Natalizumab for the treatment of active Crohn's disease: results of the ENCORE Trial. Gastroenterology 2007;132:1672-1683
40. Sandborn WJ, Colombel JF, Enns R, et al. International Efficacy of Natalizumab as Active Crohn's Therapy (ENACT-1) Trial Group; Evaluation of Natalizumab as Continuous Therapy (ENACT-2) Trial Group. Natalizumab induction and maintenance therapy for Crohn's disease. N Engl J Med 2005;353:1912-1925
41. Kleinschmidt-DeMasters BK, Tyler KL. Progressive multifocal leukoencephalopathy complicating treatment with natalizumab and interferon beta-1a for multiple sclerosis. N Engl J Med 2005;353:369-374
42. Yousry TA, Major EO, Ryschkewitsch C, et al. Evaluation of patients treated with natalizumab for progressive multifocal leukoencephalopathy. N Engl J Med 2006;354:924-933
43. Conway D, Cohen JA. Combination therapy in multiple sclerosis. Lancet Neurol 2010;9:299-308
44. Goodman AD, Rossman H, Bar-OR A, et al. GLANCE: results of a phase 2, randomized, double-blind, placebo-controlled study. Neurology 2009;72:806-812
45. Brain disease virus found in natalizumab treated MS patients. Disabled World, Montreal, Canada; 2009. Available from: http://www.disabled-world.com/ health/ autoimmunediseases/ms/natalizumab-ms. php. [Last accessed 29 June 2010]
46. Langer-Gould A, Atlas SW, Green AJ, et al. Progressive multifocal leukoencephalopathy in a patient treated with natalizumab. N Engl J Med 2005;353:375-381
47. "News Detail: National MS Society," Update on Tysabri and PML. Available from: http://www. nationalmssociety.org/news/news-detail/ index.aspx?nid=2308. [Accessed 19 August 2009]
48. Clifford DB. DeLuca A, Simpson DM, et al. Natalizumab-associated progressive multifocal leukoencephalopahty in patients with multiple sclerosis: lesions from 28 cases. Lancet Neurol 2010;9:438-446
49. Jeffery S. New case of PML with natalizumab monotherapy. Medscape, New York, NY; 17 December 2008. Available from: http://www. medscape.com/ viewarticle/585512. [Last accessed 29 June 2010]
50. Major EO. Reemergence of PML in natalizumab-treated patients-new cases, same concerns. N Engl J Med 2009;36:1041-1043
51. FDA MedWatch-Tysabri (natalizumab)-two new cases of progressive multifocal leukoencephalopathy in European patients receiving drug as monotherapy for multiple sclerosis for more than one year. Infectious Disease Society of America, Arlington, VA; Available from: http://www.idsociety. org/Content.aspx?id=11872. [Last accessed 29 June 2010]
52. New cases of PML found in MS patients taking natalizumab. American Academy of Neurology, Saint Paul, MN; 2008. Available from: http://www.aan. com/news/?event=read&article-id=5753. [Last accessed 29 June 2010]
53. Talan J. Two more patients on natalizumab develop PML. Neurol Today 2008;8:19
54. FDA Continues to Receive Reports of PML in Patients Receiving Natalizumab. US Food and Drug Administration, Rockville, MD; 2009. Available from: http://www.docguide. com/news/content.nsf/news/ 852576140048867A85257639006795F6. [Last accessed 29 June 2010]
55. Food and Drug Administration. FDA Drug Safety communication: risk of progressive multifocal leukoencephalopathy (PML) with the use of Tysabri (natalizumab). US Food and Drug Administration, Rockville, MD; Updated 2/5/2010. Available from: http://www.fda.gov/Drugs/DrugSafety/ PostmarketDrugSafetyInformationfor PatientsandProviders/ucm199872.htm. [Last accessed 29 June 2010]
56. Pocock N. FDA Medwatch: risk of PML with natalizumab increases with number of infusions. US Food and Drug Administration, Rockville, MD; 56. Available from: http://www. nelm-web.lbi.co.uk/en/NeLM-Area/ News/2010-February/08/ FDA-Medwatch-Risk-of-PML-with- natalizumab-increases- with-number-of- infusions/ [Last accessed 29 June 2010]
57. Anderson P. AAN 2009: Incidence of PML with natalizumab in MS lower than previously thought. Medscape, New York, NY; 2009. Available from: http://www.medscape.com/viewarticle/ 702373. [Last accessed 29 June 2010]
58. Jeffrey S. PML risk increases with repeated Natalizumab infusions: FDA. Medscape, New York, NY; 2010. Available from: http://www. medscape.com/ viewarticle/716536. [Last accessed 29 June 2010]
59. FDA warns of link between natalizumab, brain infection risk of PML increases as number of infusions received increases. Modern Medicine, Woodland Hills, CA; 2010. Available from: http://www. modernmedicine.com/modernmedicine/ Modern+Medicine+Now/FDA-Warns-of- Link-Between-Natalizumab-Brain-Infect/ ArticleNewsFeed/Article/detail/655741. [Last accessed 29 June 2010]
60. Jeffery S. Asymptomatic reactivation of JC virus found in natalizumab-treated MS patients; PML case reports released. Medscape, New York, NY; 2009. Available from: http://www. medscape.com/viewarticle/708701. [Last accessed 29 June 2010]
61. Lonergan RM, Carr MJ, Gascun C, et al. Reactivation of BK polyomavirus in patients with multiple sclerosis receiving natalizumab therapy. J Neurovirol 2009;15:351-359
62. Jilek S, Jaquiery E, Hirsch HH, et al. Immune responses to JC virus in patients with multiple sclerosis treated with natalizumab: a cross-sectional and longitudinal study. Lancet Neurol 2010:9:264-272
63. FDA to amend natalizumab label based on new cases of PML in Europe. US Food and Drug Administration, Rockville, MD; 2008. Available from: http://www. docguide.com/news/content.nsf/ news/852571020057CCF6852574 B0007050D9. [Last accessed 29 June 2010]
64. Ranshoff RM. PML risk and natalizumab: more questions than answers. Lancet Neurol 2010;9:231-233
65. Koralink IJ. Progressive multifocal leukoencephalopathy revisted: has the disease outgrown its name? Ann Neurol 2006;60:162-173
66. Calabrese LH, Mollow ES, Huang D, Ranshoff RM. Progressive multifocal pathologic patterns of disease. Arthritis Preum 2007;56:2116-2128
67. Information on Natalizumab. US Food and Drug Administration, Rockville, MD; 2009. Available from: http://www.fda.gov/Drugs/DrugSafety/ PostmarketDrugSafetyInformationfor PatientsandProviders/ucm107198.htm. [Last accessed 29 June 2010]
68. EMEA recommends update of product information of natalizumab and risk of progressive multifocal leukoencephalopathy (PML). European Medicines Agency, London, UK; 2008. Available from: http://www.ema. europa.eu/humandocs/PDFs/EPAR/ tysabri/49661208en.pdf. [Last accessed 29 June 2010]
69. Tysabri (natalizumab) Marketing Suspended. US Food and Drug Administration, Rockville, MD; 2005. Available from: http://www. medicinenet.com/script/main/art. asp?articlekey=47298. [Last accessed 29 June 2010]
70. US FDA issues alert to healthcare professionals regarding natalizumab and progressive multifocal leukoencephalopathy. US Food and Drug Administration, Rockville, MD; 2008. Available from: http://www. nelm.nhs.uk/en/NeLM-Area/News/ 2008-August/26/US-FDA-issues- alert-to-healthcare-professionals- regarding-natalizumab-and-progressive- multifocal-leukoencephalopathy/ [Last accessed 29 June 2010]
71. Hanauer SB, Feagan BG, Lichtenstein GR, et al. Maintenance infliximab for Crohn's disease: the ACCENT I randomized trial. Lancet 2002;359:1541-1549
72. Colombel JF, Sandborn WJ, Rutgeerts P, et al. Adalimumab for maintenance of clinical response and remission in patients with Crohn's disease: the CHARM trial. Gastroenterolgy 2007;132:52-65
73. Tysabri Prescribing Information, Biogen Idec, Inc., 2008. [Update] Available from: http://www.accessdata. fda.gov/drugsatfda-docs/label/2008/ 125104s0067lbl.pdf 59.