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2. Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study

Author

Deegan, Patrick B., Goker-Alpan, Ozlem, Geberhiwot, Tarekegn, Hopkin, Robert J., Lukina, Elena, Tylki-Szymanska, Anna, Zaher, Atef, Sensinger, Charlotte, Gaemers, Sebastiaan J.M., Modur, Vijay, Thurberg, Beth L., Sharma, Jyoti, Najafian, Behzad, Mauer, Michael, DasMahapatra, Pronabesh, Wilcox, William R., and Germain, Dominique P.

Published
2023
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4. Fabry patient's experience of pegunigalsidase alfa monthly infusion: PEOPLE study

Author

Mehta, Ankit, primary, Eyskens, Francois J., additional, Pisani, Antonio, additional, Feldt-Rasmussen, Ulla, additional, Hughes, Derralynn, additional, Deegan, Patrick B., additional, Goker-Alpan, Ozlem, additional, Wallace, Eric, additional, Bernat, John, additional, Longo, Nicola, additional, Bianchi, Francesco, additional, Rocco, Rossana, additional, and Paleari, Ylenia, additional

Published
2024
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5. Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study

Author

Wallace, Eric L, primary, Goker-Alpan, Ozlem, additional, Wilcox, William R, additional, Holida, Myrl, additional, Bernat, John, additional, Longo, Nicola, additional, Linhart, Aleš, additional, Hughes, Derralynn A, additional, Hopkin, Robert J, additional, Tøndel, Camilla, additional, Langeveld, Mirjam, additional, Giraldo, Pilar, additional, Pisani, Antonio, additional, Germain, Dominique Paul, additional, Mehta, Ankit, additional, Deegan, Patrick B, additional, Molnar, Maria Judit, additional, Ortiz, Damara, additional, Jovanovic, Ana, additional, Muriello, Michael, additional, Barshop, Bruce A, additional, Kimonis, Virginia, additional, Vujkovac, Bojan, additional, Nowak, Albina, additional, Geberhiwot, Tarekegn, additional, Kantola, Ilkka, additional, Knoll, Jasmine, additional, Waldek, Stephen, additional, Nedd, Khan, additional, Karaa, Amel, additional, Brill-Almon, Einat, additional, Alon, Sari, additional, Chertkoff, Raul, additional, Rocco, Rossana, additional, Sakov, Anat, additional, and Warnock, David G, additional

Published
2023
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6. Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study.

Author

Wallace, Eric L., Goker-Alpan, Ozlem, Wilcox, William R., Holida, Myrl, Bernat, John, Longo, Nicola, Linhart, Aleš, Hughes, Derralynn A., Hopkin, Robert J., Tøndel, Camilla, Langeveld, Mirjam, Giraldo, Pilar, Pisani, Antonio, Germain, Dominique Paul, Mehta, Ankit, Deegan, Patrick B., Molnar, Maria Judit, Ortiz, Damara, Jovanovic, Ana, and Muriello, Michael

Abstract

Background Pegunigalsidase alfa is a PEGylated a-galactosidase A enzyme replacement therapy. BALANCE (NCT02795676) assessed non-inferiority of pegunigalsidase alfa versus agalsidase beta in adults with Fabry disease with an annualised estimated glomerular filtration rate (eGFR) slope more negative than -2 mL/min/1.73 m2/year who had received agalsidase beta for =1 year. Methods Patients were randomly assigned 2:1 to receive 1 mg/kg pegunigalsidase alfa or agalsidase beta every 2 weeks for 2 years. The primary efficacy analysis assessed non-inferiority based on median annualised eGFR slope differences between treatment arms. Results Seventy-seven patients received either pegunigalsidase alfa (n=52) or agalsidase beta (n=25). At baseline, mean (range) age was 44 (18-60) years, 47 (61%) patients were male, median eGFR was 74.5 mL/min/1.73 m2 and median (range) eGFR slope was -7.3 (-30.5, 6.3) mL/min/1.73 m2/year. At 2 years, the difference between median eGFR slopes was -0.36 mL/min/1.73 m2/year, meeting the prespecified non-inferiority margin. Minimal changes were observed in lyso-Gb3 concentrations in both treatment arms at 2 years. Proportions of patients experiencing treatment-related adverse events and mild or moderate infusion-related reactions were similar in both groups, yet exposure-adjusted rates were 3.6-fold and 7.8-fold higher, respectively, with agalsidase beta than pegunigalsidase alfa. At the end of the study, neutralising antibodies were detected in 7 out of 47 (15%) pegunigalsidase alfa-treated patients and 6 out of 23 (26%) agalsidase beta-treated patients. There were no deaths. Conclusions Based on rate of eGFR decline over 2 years, pegunigalsidase alfa was non-inferior to agalsidase beta. Pegunigalsidase alfa had lower rates of treatment-emergent adverse events and mild or moderate infusion-related reactions. [ABSTRACT FROM AUTHOR]

Published
2024
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9. Long-term safety and efficacy of pegunigalsidase alfa administered every 4 weeks in patients with Fabry disease:Two-year interim results from the ongoing phase 3 BRIGHT51 open-label extension study

Author

Bernat, John, Holida, Myrl D., Longo, Nicola, Goker-alpan, Ozlem, Wallace, Eric, Deegan, Patrick B., Tøndel, Camilla, Eyskens, Francois J., Feldt-rasmussen, Ulla, Hughes, Derralynn, Pisani, Antonio, Rocco, Rossana, Almon, Einat Brill, Alon, Sari, Chertkoff, Raul, Warnock, David G., Waldek, Stephen, Wilcox, William R., Bernat, John, Holida, Myrl D., Longo, Nicola, Goker-alpan, Ozlem, Wallace, Eric, Deegan, Patrick B., Tøndel, Camilla, Eyskens, Francois J., Feldt-rasmussen, Ulla, Hughes, Derralynn, Pisani, Antonio, Rocco, Rossana, Almon, Einat Brill, Alon, Sari, Chertkoff, Raul, Warnock, David G., Waldek, Stephen, and Wilcox, William R.

Abstract

Fabry disease (FD) is an inherited disorder caused by alpha-galactosidase-A deficiency leading to accumulation of globotriaosylceramide (Gb3), its metabolite lyso-Gb3, and other sphingolipids, resulting in impaired organ function. Current treatments include enzyme replacement therapies (ERTs) requiring infusions every 2 weeks (Q2W). Pegunigalsidase alfa (PA) is a PEGylated alpha-galactosidase-A enzyme with increased half-life compared with current ERTs, potentially allowing for dosing flexibility (1.0 mg/kg Q2W or 2.0 mg/kg every 4 weeks [Q4W]). BRIGHT51 (PB-102-F51, NCT03614234), an ongoing phase 3, open-label extension, evaluates the safety and efficacy of 2.0 mg/kg PA Q4W in adults with FD up to 4 years. Patients were eligible if they had completed BRIGHT, a 1-year switchover clinical trial for patients who previously received agalsidase alfa or agalsidase beta Q2W. For this interim analysis, safety and efficacy are reported from baseline in BRIGHT to ≥2 years of treatment. 29 adults (23 M:6F; mean age 40.9 years) were enrolled in BRIGHT51, with mean (range) PA exposure of 38.3 (25.3–44.8) person-months. 27/29 patients (93.1%) reported 339 treatment-emergent adverse events (TEAEs); 11/29 patients (37.9%) reported 46 PA-related TEAEs, of which none were serious/severe. 6/29 patients (20.7%, all males) experienced a total of 38 infusion-related reactions, all mild/moderate in intensity. Nine patients had antidrug antibodies (ADAs) at baseline; no de novo ADAs developed following the switch to PA for ≥2 years. At week 108, mean (SE) change from baseline of eGFR was −5.10 (1.96) mL/min/1.73m2; overall, the mean (SE) eGFR slope was −2.77 (0.54) mL/min/1.73m2/year (male: −3.03 [0.61] mL/min/1.73m2/year; female: -1.74 [1.21] mL/min/1.73m2/year). Mean (SE) plasma lyso-Gb3 was stable through week 108 (baseline: 19.36 [3.35] nmol/L; week 108: 22.98 [3.72] nmol/L). PA 2.0 mg/kg Q4W showed no new safety concerns during ≥2 years; additional analyses are needed to further eval

Published
2023

11. Long-term safety and efficacy of pegunigalsidase alfa administered every 4 weeks in patients with Fabry disease: Two-year interim results from the ongoing phase 3 BRIGHT51 open-label extension study

Author

Bernat, John, primary, Holida, Myrl D., additional, Longo, Nicola, additional, Goker-Alpan, Ozlem, additional, Wallace, Eric, additional, Deegan, Patrick B., additional, Tøndel, Camilla, additional, Eyskens, Francois J., additional, Feldt-Rasmussen, Ulla, additional, Hughes, Derralynn, additional, Pisani, Antonio, additional, Rocco, Rossana, additional, Almon, Einat Brill, additional, Alon, Sari, additional, Chertkoff, Raul, additional, Warnock, David G., additional, Waldek, Stephen, additional, and Wilcox, William R., additional

Published
2023
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