Association of immune response with efficacy and safety outcomes in adults with phenylketonuria administered pegvaliase in phase 3 clinical trials

EBioMedicine - Tập 37 - Trang 366-373 - 2018
Soumi Gupta1, Kelly Lau1, Cary O. Harding2, Gillian Shepherd3, Ryan Boyer1, John P. Atkinson4, Vijaya Knight5, Joy Olbertz1, Kevin Larimore1, Zhonghu Gu1, Mingjin Li1, Orli Rosen1, Stephen J. Zoog1, Haoling H. Weng1, Becky Schweighardt1
1BioMarin Pharmaceutical Inc., 105 Digital Dr, Novato, CA 94949, United States
2Oregon Health & Science University, 3181 SW Sam Jackson Park Rd, Portland, OR 97239, United States
3Weill Medical College of Cornell University, 47 East 91st St, New York, NY 10128, United States
4Washington University School of Medicine, Campus Box 8045, 660 S. Euclid Ave, St. Louis, MO 63110, United States
5National Jewish Health Advanced Diagnostic Laboratories, 1400 Jackson St, Denver, CO 80206, United States

Tài liệu tham khảo

Bik-Multanowski, 2008, Quality of life in noncompliant adults with phenylketonuria after resumption of the diet, J Inherit Metab Dis, 31, S415, 10.1007/s10545-008-0978-7 Enns, 2010, Suboptimal outcomes in patients with PKU treated early with diet alone: revisiting the evidence, Mol Genet Metab, 101, 99, 10.1016/j.ymgme.2010.05.017 Bilder, 2017, Neuropsychiatric comorbidities in adults with phenylketonuria: a retrospective cohort study, Mol Genet Metab, 121, 1, 10.1016/j.ymgme.2017.03.002 Thomas, 2018, Pegvaliase for the treatment of phenylketonuria: results of a long-term phase 3 clinical trial program (PRISM), Mol Genet Metab, 124, 27, 10.1016/j.ymgme.2018.03.006 Brown, 2016, Phenylketonuria (PKU): A problem solved?, Mol Genet Metab Rep, 6, 8, 10.1016/j.ymgmr.2015.12.004 Vockley, 2014, Phenylalanine hydroxylase deficiency: diagnosis and management guideline, Genet Med, 16, 188, 10.1038/gim.2013.157 Blau, 2015, Alternative therapies to address the unmet medical needs of patients with phenylketonuria, Expert Opin Pharmacother, 16, 791, 10.1517/14656566.2015.1013030 Jurecki, 2017, Adherence to clinic recommendations among patients with phenylketonuria in the United States, Mol Genet Metab, 120, 190, 10.1016/j.ymgme.2017.01.001 Koch, 2002, Phenylketonuria in adulthood: a collaborative study, J Inherit Metab Dis, 25, 333, 10.1023/A:1020158631102 BioMarin Pharmaceutical Inc Pegvaliase (Palynziq): US prescribing information; 2018. https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/761079s000lbl.pdf (accessed July 10, 2018). Koren, 2008, Recommendations on risk-based strategies for detection and characterization of antibodies against biotechnology products, J Immunol Methods, 333, 1, 10.1016/j.jim.2008.01.001 Wang, 2014, Immunogenicity and PK/PD evaluation in biotherapeutic drug development: scientific considerations for bioanalytical methods and data analysis, Bioanalysis, 6, 79, 10.4155/bio.13.302 Krishna, 2016, Immunogenicity to biotherapeutics - the role of anti-drug immune complexes, Front Immunol, 7, 21, 10.3389/fimmu.2016.00021 Ponce, 1997, Enzyme therapy in Gaucher disease type 1: effect of neutralizing antibodies to acid beta-glucosidase, Blood, 90, 43, 10.1182/blood.V90.1.43 Chirmule, 2012, Immunogenicity to therapeutic proteins: impact on PK/PD and efficacy, AAPS J, 14, 296, 10.1208/s12248-012-9340-y Verhoef, 2013, Questioning the use of PEGylation for drug delivery, Drug Deliv Transl Res, 3, 499, 10.1007/s13346-013-0176-5 Verhoef, 2014, Potential induction of anti-PEG antibodies and complement activation toward PEGylated therapeutics, Drug Discov Today, 19, 1945, 10.1016/j.drudis.2014.08.015 Hershfield, 2014, Induced and pre-existing anti-polyethylene glycol antibody in a trial of every 3-week dosing of pegloticase for refractory gout, including in organ transplant recipients, Arthritis Res Ther, 16, R63, 10.1186/ar4500 Wang, 2008, Structural and biochemical characterization of the therapeutic Anabaena variabilis phenylalanine ammonia lyase, J Mol Biol, 380, 623, 10.1016/j.jmb.2008.05.025 Medical Dictionary for Regulatory Activities (MedDRA) Sampson, 2006, Second symposium on the definition and management of anaphylaxis: summary report--second National Institute of Allergy and Infectious Disease/Food Allergy and Anaphylaxis Network symposium, Ann Emerg Med, 47, 373, 10.1016/j.annemergmed.2006.01.018 Brown, 2004, Clinical features and severity grading of anaphylaxis, J Allergy Clin Immunol, 114, 371, 10.1016/j.jaci.2004.04.029 National Cancer Institute Yang, 2016, Analysis of pre-existing IgG and IgM antibodies against polyethylene glycol (PEG) in the general population, Anal Chem, 88, 11804, 10.1021/acs.analchem.6b03437 Chen, 2016, Measurement of pre-existing IgG and IgM antibodies against polyethylene glycol in healthy individuals, Anal Chem, 88, 10661, 10.1021/acs.analchem.6b03109 Nguyen, 2018, Depletion of IgG and IgM before IgE assay indicates pegvaliase-induced hypersensitivity reactions are not IgE-mediated Lubich, 2016, The mystery of antibodies against polyethylene glycol (PEG) - what do we know?, Pharm Res, 33, 2239, 10.1007/s11095-016-1961-x Ishida, 2006, Injection of PEGylated liposomes in rats elicits PEG-specific IgM, which is responsible for rapid elimination of a second dose of PEGylated liposomes, J Control Release, 112, 15, 10.1016/j.jconrel.2006.01.005 Yang, 2015, Anti-PEG immunity: emergence, characteristics, and unaddressed questions, Wiley Interdiscip Rev Nanomed Nanobiotechnol, 7, 655, 10.1002/wnan.1339