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Pegvaliase for the treatment of phenylketonuria: Results of a long-term phase 3 clinical trial program (PRISM).
Thomas J, Levy H, Amato S, Vockley J, Zori R, Dimmock D, Harding CO, Bilder DA, Weng HH, Olbertz J, Merilainen M, Jiang J, Larimore K, Gupta S, Gu Z, Northrup H; PRISM investigators. Thomas J, et al. Mol Genet Metab. 2018 May;124(1):27-38. doi: 10.1016/j.ymgme.2018.03.006. Epub 2018 Mar 31. Mol Genet Metab. 2018. PMID: 29653686 Free article. Clinical Trial.
In PRISM-1, pegvaliase-naive participants with blood Phe >600 mumol/L were randomized 1:1 to a maintenance dose of 20 mg/day or 40 mg/day of pegvaliase. Participants in PRISM-1 continued pegvaliase treatment in PRISM-2, a 4-part clinical trial that include …
In PRISM-1, pegvaliase-naive participants with blood Phe >600 mumol/L were randomized 1:1 to a maintenance dose of 20 mg/day or 40 …
Pegvaliase: a novel treatment option for adults with phenylketonuria.
Mahan KC, Gandhi MA, Anand S. Mahan KC, et al. Curr Med Res Opin. 2019 Apr;35(4):647-651. doi: 10.1080/03007995.2018.1528215. Epub 2018 Oct 25. Curr Med Res Opin. 2019. PMID: 30247930 Review.
RESULTS: Pegvaliase is effective in decreasing blood phenylalanine levels, and is associated with a manageable side-effect profile. ...Brief sub-studies also showed the improvement in inattention symptoms in patients treated with pegvaliase, compared to placebo. CON …
RESULTS: Pegvaliase is effective in decreasing blood phenylalanine levels, and is associated with a manageable side-effect profile. . …
A Comprehensive Review of Pegvaliase, an Enzyme Substitution Therapy for the Treatment of Phenylketonuria.
Hydery T, Coppenrath VA. Hydery T, et al. Drug Target Insights. 2019 Jun 21;13:1177392819857089. doi: 10.1177/1177392819857089. eCollection 2019. Drug Target Insights. 2019. PMID: 31258325 Free PMC article. Review.
OBJECTIVE: To review the pharmacology, pharmacokinetics, efficacy, safety, and place in therapy of a phenylalanine-metabolizing enzyme indicated to reduce blood phenylalanine concentrations, pegvaliase injection. ...DATA SYNTHESIS: Pegvaliase is a pegylated P …
OBJECTIVE: To review the pharmacology, pharmacokinetics, efficacy, safety, and place in therapy of a phenylalanine-metabolizing enzym …
Pegvaliase: First Global Approval.
Markham A. Markham A. BioDrugs. 2018 Aug;32(4):391-395. doi: 10.1007/s40259-018-0292-3. BioDrugs. 2018. PMID: 30022433
Based on these positive results pegvaliase was recently approved in the US for adults with phenylketonuria who have uncontrolled blood phenylalanine concentrations on current treatment. This article summarizes the milestones in the development of pegvaliase leading …
Based on these positive results pegvaliase was recently approved in the US for adults with phenylketonuria who have uncontrolled bloo …
Achieving efficacy in subjects with sustained pegvaliase-neutralizing antibody responses.
Aryal M, Lau K, Boyer R, Zhou H, Abend J, Gu K, Olbertz J, Gupta S, Zoog S, Larimore K. Aryal M, et al. Mol Genet Metab. 2021 Nov;134(3):235-242. doi: 10.1016/j.ymgme.2021.09.006. Epub 2021 Oct 4. Mol Genet Metab. 2021. PMID: 34716085 Free article. Clinical Trial.
In Phase 3 clinical studies, all subjects treated with pegvaliase developed anti-drug antibodies. To specifically evaluate pegvaliase-neutralizing antibodies (NAbs) and assess impact on pegvaliase efficacy, a novel hybrid ligand-binding/tandem mass spectromet …
In Phase 3 clinical studies, all subjects treated with pegvaliase developed anti-drug antibodies. To specifically evaluate pegvali …
Pegvaliase: Immunological profile and recommendations for the clinical management of hypersensitivity reactions in patients with phenylketonuria treated with this enzyme substitution therapy.
Hausmann O, Daha M, Longo N, Knol E, Müller I, Northrup H, Brockow K. Hausmann O, et al. Mol Genet Metab. 2019 Sep-Oct;128(1-2):84-91. doi: 10.1016/j.ymgme.2019.05.006. Epub 2019 Jun 17. Mol Genet Metab. 2019. PMID: 31375398 Free article. Review.
METHODS: Eight European academic immunology experts with a broad range of experience in hypersensitivity, anaphylaxis, and/or drug reactions, and two geneticists from the USA with pegvaliase experience convened for two advisory board meetings. Efficacy, safety, and immunol …
METHODS: Eight European academic immunology experts with a broad range of experience in hypersensitivity, anaphylaxis, and/or drug reactions …
Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial.
Harding CO, Amato RS, Stuy M, Longo N, Burton BK, Posner J, Weng HH, Merilainen M, Gu Z, Jiang J, Vockley J; PRISM-2 Investigators. Harding CO, et al. Mol Genet Metab. 2018 May;124(1):20-26. doi: 10.1016/j.ymgme.2018.03.003. Epub 2018 Mar 18. Mol Genet Metab. 2018. PMID: 29628378 Free article. Clinical Trial.
METHODS: PRISM-2 is a 4-part, Phase 3 study that enrolled adults with PKU receiving pegvaliase treatment (initiated in a prior Phase 2 or Phase 3 study). ...RESULTS: The pooled pegvaliase group enrolled 66 participants and each placebo group enrolled 14 participants …
METHODS: PRISM-2 is a 4-part, Phase 3 study that enrolled adults with PKU receiving pegvaliase treatment (initiated in a prior Phase …
Nutrition status of adults with phenylketonuria treated with pegvaliase.
Viau K, Wessel A, Martell L, Sacharow S, Rohr F. Viau K, et al. Mol Genet Metab. 2021 Aug;133(4):345-351. doi: 10.1016/j.ymgme.2021.06.002. Epub 2021 Jun 7. Mol Genet Metab. 2021. PMID: 34116943
BACKGROUND: Pegvaliase is an enzyme substitution therapy that reduces blood phenylalanine (Phe) in adults with phenylketonuria (PKU), and often allows normalization of protein intake (0.8 g protein/kg). ...RESULTS: Participants (n = 18, 61% female) started pegvaliase …
BACKGROUND: Pegvaliase is an enzyme substitution therapy that reduces blood phenylalanine (Phe) in adults with phenylketonuria (PKU), …
Depletion of interfering IgG and IgM is critical to determine the role of IgE in pegvaliase-associated hypersensitivity.
Larimore K, Nguyen T, Badillo B, Lau K, Zori R, Shepherd G, Zoog SJ, Weng HH, Gupta S. Larimore K, et al. J Immunol Methods. 2019 May;468:20-28. doi: 10.1016/j.jim.2019.03.004. Epub 2019 Mar 14. J Immunol Methods. 2019. PMID: 30880261
In phase 3 clinical studies, pegvaliase substantially reduced mean blood Phe in adult subjects with PKU. ...Taken together, the clinical presentation, temporal association of HAEs with CIC levels and complement activation, and lack of anti-pegvaliase IgE suggest …
In phase 3 clinical studies, pegvaliase substantially reduced mean blood Phe in adult subjects with PKU. ...Taken together, the clini …
Association of immune response with efficacy and safety outcomes in adults with phenylketonuria administered pegvaliase in phase 3 clinical trials.
Gupta S, Lau K, Harding CO, Shepherd G, Boyer R, Atkinson JP, Knight V, Olbertz J, Larimore K, Gu Z, Li M, Rosen O, Zoog SJ, Weng HH, Schweighardt B. Gupta S, et al. EBioMedicine. 2018 Nov;37:366-373. doi: 10.1016/j.ebiom.2018.10.038. Epub 2018 Oct 23. EBioMedicine. 2018. PMID: 30366815 Free PMC article. Clinical Trial.
Blood Phe decreased over time as CIC levels and complement activation declined and pegvaliase dosage increased. HAEs were most frequent during early treatment and declined over time. ...No evidence of pegvaliase-associated IC-mediated end organ damage was noted. INT …
Blood Phe decreased over time as CIC levels and complement activation declined and pegvaliase dosage increased. HAEs were most freque …
18 results