Lanadelumab Injection Treatment For The Prevention Of Hereditary Angioedema (HAE): Design, Development And Place In Therapy

References

• Cicardi M, Aberer W, Banerji A, et al. Classification, diagnosis and approach to treatment for angioedema: consensus report from the Hereditary Angioedema International Working Group. Allergy. 2014;69:602–616. doi:10.1111/all.1238024673465
   PubMed Web of Science ®Google Scholar
• Banerji A, Busse P, Christiansen SC, et al. Current state of hereditary angioedema management: a patient survey. Allergy Asthma Proc. 2015;36(3):213–217. doi:10.2500/aap.2015.36.382425976438
   PubMed Web of Science ®Google Scholar
• Aygören-Pürsün E, Bygum A, Beusterien K, et al. Socioeconomic burden of hereditary angioedema: results from the hereditary angioedema burden of illness study in Europe. Orphanet J Rare Dis. 2014;4(9):99. doi:10.1186/1750-1172-9-99
   Web of Science ®Google Scholar
• Bova M, De Feo G, Parente R, et al. Hereditary and acquired angioedema: heterogeneity of pathogenesis and clinical phenotypes. Int Arch Allergy Immunol. 2018;175(3):126–135. doi:10.1159/00048631229393169
   PubMed Web of Science ®Google Scholar
• Valerieva A, Perego F, Porebski G, et al. Hereditary angioedema: treatment options and availability. Balance between patients’ needs and stakeholders’ plans. Alergia Astma Immunologia. 2018;23(4).
   Web of Science ®Google Scholar
• Blankart CR, Stargardt T, Schreyögg J. Availability of and access to orphan drugs: an international comparison of pharmaceutical treatments for pulmonary arterial hypertension, fabry disease, hereditary angioedema and chronic myeloid leukaemia. Pharmacoeconomics. 2011;29(1):63–82. doi:10.2165/11539190-000000000-0000021073206
   PubMed Web of Science ®Google Scholar
• Longhurst H, Bygum A. The humanistic, societal, and pharmaco-economic burden of angioedema. Clin Rev Allergy Immunol. 2016;51(2):230–239. doi:10.1007/s12016-016-8575-227388236
   PubMed Web of Science ®Google Scholar
• Federici C, Perego F, Borsoi L, et al. Costs and effects of on-demand treatment of hereditary angioedema in Italy: a prospective cohort study of 167 patients. BMJ Open. 2018;8:e022291. doi:10.1136/bmjopen-2018-022291
   PubMed Web of Science ®Google Scholar
• Aygören-Pürsün E, Magerl M, Maetzel A, et al. Epidemiology of Bradykinin-mediated angioedema: a systematic investigation of epidemiological studies. Orphanet J Rare Dis. 2018;13(1):73. doi:10.1186/s13023-018-0822-629728119
   PubMed Web of Science ®Google Scholar
• Dewald G, Bork K. Missense mutations in the coagulation factor XII (Hageman factor) gene in hereditary angioedema with normal C1 inhibitor. Biochem Biophys Res Commun. 2006;343(4):1286–1289. doi:10.1016/j.bbrc.2006.03.09216638441
   PubMed Web of Science ®Google Scholar
• Bafunno V, Firinu D, D’Apolito M, et al. Hereditary angioedema with a mutation in the plasminogen gene. J Allergy Clin Immunol. 2017;S0091-6749(17): 30921–1.
   Google Scholar
• Bork K, Wulff K, Steinmüller-Magin L, et al. Hereditary angioedema cosegregating with a novel kininogen1 gene mutation changing the N-terminal cleavage site of bradykinin. Allergy. 2018;73(2):442–450. doi:10.1111/all.1327028795768
   PubMed Web of Science ®Google Scholar
• Bork K, Wulff K, Rossmann H, et al. Hereditary angioedema cosegregating with a novel kininogen 1 gene mutation changing the N-terminal cleavage site of bradykinin. Allergy. 2019. doi:10.1111/all.13869
   Web of Science ®Google Scholar
• Caccia S, Suffritti C, Cicardi M. Pathophysiology of hereditary angioedema. Pediatr Allergy Immunol Pulmonol. 2014;27(4):159–163. doi:10.1089/ped.2014.042525538858
   PubMed Web of Science ®Google Scholar
• Cicardi M, Banerji A, Bracho F, et al. Icatibant, a new bradykinin-receptor antagonist, in hereditary angioedema. N Engl J Med. 2010;363(6):532–541. doi:10.1056/NEJMoa090639320818888
   PubMed Web of Science ®Google Scholar
• Bork K, Wulff K, Hardt J, et al. Hereditary angioedema caused by missense mutations in the factor XII gene: clinical features, trigger factors, and therapy. J Allergy Clin Immunol. 2009;124:129–134. doi:10.1016/j.jaci.2009.03.03819477491
   PubMed Web of Science ®Google Scholar
• Bjorkqvist J, de Maat S, Lewandrowski U, et al. Defective glycosylation of coagulation factor XII underlies hereditary angioedema type III. J Clin Invest. 2015;125:3132–3146. doi:10.1172/JCI7713926193639
   PubMed Web of Science ®Google Scholar
• Bafunno V, Firinu D, D’Apolito M, et al. Mutation of the angiopoietin-1 gene (ANGPT1) associates with a new type of hereditary angioedema. J Allergy Clin Immunol. 2018;141:1009–1017. doi:10.1016/j.jaci.2017.05.02028601681
   PubMed Web of Science ®Google Scholar
• Bork K, Wulff K, Steinmüller-Magin L, et al. Hereditary angioedema with a mutation in the plasminogen gene. Allergy. 2018;73:442–450. doi:10.1111/all.1327028795768
   PubMed Web of Science ®Google Scholar
• Li HH, Riedl M, Kashkin J. Update on the use of C1-esterase inhibitor replacement therapy in the acute and prophylactic treatment of hereditary angioedema. Clin Rev Allergy Immunol. 2019;56:207–218. doi:10.1007/s12016-018-8684-129909591
   PubMed Web of Science ®Google Scholar
• Cicardi M, Suffritti C, Perego F, Caccia S. Novelties in the diagnosis and treatment of angioedema. J Investig Allergol Clin Immunol. 2016;26(4):212–221. doi:10.18176/jiaci.0087
   PubMed Web of Science ®Google Scholar
• Riedl MA. Critical appraisal of androgen use in hereditary angioedema: a systematic review. Ann Allergy Asthma Immunol. 2015;114(4):281–288. doi:10.1016/j.anai.2015.01.00325707325
   PubMed Web of Science ®Google Scholar
• Riedl MA, Grivcheva-Panovska V, Moldovan D, et al. Recombinant human C1 esterase inhibitor for prophylaxis of hereditary angio-oedema: a phase 2, multicentre, randomised, double-blind, placebo-controlled crossover trial. Lancet. 2017;390(10102):1595–1602. doi:10.1016/S0140-6736(17)31963-328754491
   PubMed Web of Science ®Google Scholar
• Aygören-Pürsün E, Bygum A, Grivcheva-Panovska V, et al. Oral plasma kallikrein inhibitor for prophylaxis in hereditary angioedema. N Engl J Med. 2018;379(4):352–362. doi:10.1056/NEJMoa171699530044938
   PubMed Web of Science ®Google Scholar
• Perego F, Wu MA, Valerieva A, et al. Current and emerging biologics for the treatment of hereditary angioedema. Expert Opin Biol Ther. 2019;19(6):517–526. doi:10.1080/14712598.2019.159558130912460
   PubMed Web of Science ®Google Scholar
• Liu J, Qin J, Borodovsky A, et al. An investigational RNAi therapeutic targeting factor XII (ALN-F12) for the treatment of hereditary angioedema. RNA. 2019;25(2):255–263. doi:10.1261/rna.068916.11830463937
   PubMed Web of Science ®Google Scholar
• Qiu T, Chiuchiolo MJ, Whaley AS, et al. Gene therapy for C1 esterase inhibitor deficiency in a Murine model of hereditary angioedema. Allergy. 2019;74(6):1081–1089. doi:10.1111/all.1358230059156
   PubMed Web of Science ®Google Scholar
• Bhattacharjee G, Revenko AS, Crosby JR, et al. Inhibition of vascular permeability by antisense-mediated inhibition of plasma kallikrein and coagulation factor 12. Nucleic Acid Ther. 2013;23(3):175–187. doi:10.1089/nat.2013.041723582057
   PubMed Web of Science ®Google Scholar
• Kenniston JA, Faucette RR, Martik D, et al. Inhibition of plasma kallikrein by a highly specific active site blocking antibody. J Biol Chem. 2014;289(34):23596–23608. doi:10.1074/jbc.M114.56906124970892
   PubMed Web of Science ®Google Scholar
• Hoet RM, Cohen EH, Kent RB, et al. Generation of high-affinity human antibodies by combining donor-derived and synthetic complementarity-determining-region diversity. Nat Biotechnol. 2005;23:344–348.15723048
   PubMed Web of Science ®Google Scholar
• Chyung Y, Vince B, Iarrobino R, et al. A phase 1 study investigating DX-2930 in healthy subjects. Ann Allergy Asthma Immunol. 2014;113(4):460–466. doi:10.1016/j.anai.2014.05.02824980392
   PubMed Web of Science ®Google Scholar
• Banerji A, Busse P, Shennak M, et al. Inhibiting Plasma Kallikrein for hereditary angioedema prophylaxis. N Engl J Med. 2017;376(8):717–728. doi:10.1056/NEJMoa160576728225674
   PubMed Web of Science ®Google Scholar
• Banerji A, Riedl MA, Bernstein JA, et al. Effect of lanadelumab compared with placebo on prevention of hereditary angioedema attacks: a randomized clinical trial. JAMA. 2018;320(20):2108–2121. doi:10.1001/jama.2018.1677330480729
   PubMed Web of Science ®Google Scholar
• Riedl MA, Bernstein JA, Craig T, et al. An open-label study to evaluate the long-term safety and efficacy of lanadelumab for prevention of attacks in hereditary angioedema: design of the HELP study extension. Clin Transl Allergy. 2017;7:36. doi:10.1186/s13601-017-0172-929043014
   PubMed Web of Science ®Google Scholar
• Wang Y, Marier J, Kassir N, Gosselin NH, Martin P. Exposure-response analyses of lanadelumab in patients with hereditary angioedema. J Allergy Clin Immunol. 2019;143(2):AB40. doi:10.1016/j.jaci.2018.12.122
   Web of Science ®Google Scholar
• Banerji A, Manning ME, Martinez-Saguer I, et al. Subgroup analyses from the Phase 3 HELP study of lanadelumab for the prevention of hereditary angioedema attacks. J Allergy Clin Immunol. 2019;143(2):AB42. doi:10.1016/j.jaci.2018.12.126
   Web of Science ®Google Scholar
• Inhaber N, Zuraw BL, Marier J, et al. Relationship between body weight, PK/ PD, and attack responses following subcutaneous administration of lanadelumab in patients with hereditary angioedema. J Allergy Clin Immunol. 2019;143(2):AB38. doi:10.1016/j.jaci.2018.12.117
   Web of Science ®Google Scholar
• Riedl MA, Anderson JT, Cicardi M, et al. Efficacy of lanadelumab in hereditary angioedema patients switching from C1 inhibitor long-term prophylaxis: interim results from the HELP open-label extension study. J Allergy Clin Immunol. 2018;143(2):AB37. doi:10.1016/j.jaci.2018.11.043
   Web of Science ®Google Scholar
• Jacobs JS, Bernstein J, Davis-Lorton M, et al. Increased response to higher dose lanadelumab in hereditary angioedema patients: exploratory findings from the HELP and HELP OLE studies. J Allergy Clin Immunol. 2019;143(2):AB38. doi:10.1016/j.jaci.2018.12.116
   Web of Science ®Google Scholar
• Busse PJ, Tachdjian R, Martinez-Saguer I, et al. Efficacy and safety of lanadelumab for prophylactic treatment in adolescents with hereditary angioedema (HAE). J Allergy Clin Immunol. 143(2):AB43. doi:10.1016/j.jaci.2018.12.131
   Web of Science ®Google Scholar
• Konrad Bork Günther Witzke. Shortened activated partial thromboplastin time may help in diagnosing hereditary and acquired angioedema. Int Arch Allergy Immunol. 2016;170:101–107. doi:10.1159/00044769527463190
   PubMed Web of Science ®Google Scholar
• Schmaier AH, Bauer KA, Cicardi M, et al. Effect of lanadelumab on coagulation parameters in patients with hereditary angioedema: findings from the phase 3 HELP study. AAAI 2019 San Francisco Poster.
   Google Scholar
• Sexton DJ, Brown N, Lumry W, et al. Lanadelumab and cardiovascular risk: findings from the phase 3 HELP study. AAAI 2019 San Francisco Poster.
   Google Scholar
• Available from: https://www.int.takhzyro.com/downloads/pdf/TAKHZYRO_International_EU_SmPC_November_2018.pdf.
   Google Scholar
• Hathaway WE, Belhasen LP, Hathaway HS. Evidence for a new plasma thromboplastin factor. 1. Case report, coagulation studies, and physicochemical properties. Blood. 1965;26:521–532.5845778
   PubMed Web of Science ®Google Scholar
• Saito H. Studies on Fletcher trait and Fitzgerald trait. A rare chance to disclose body’s defense reactions against injury. Thromb Haemost. 2010;104(05):867–874. doi:10.1160/TH10-01-005820838736
   PubMed Web of Science ®Google Scholar
• Valerieva A, Petkova E, Suffritti C Rare forms of angioedema in bulgaria: experience since 1972. Past And Present EAACI 2019, Lisbon, Poster.
   Google Scholar