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Table 2 Reveglucosidase alfa mean (range) PK parameters in LOPD subjectsa

From: A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease

  5 mg/kg 10 mg/kg 20 mg/kg
  Day 1 Week 12 Week 24 Day 1 Week 12 Week 24 Day 1 Week 12 Week 24
Mean PK parameter (n = 3) (n = 3) (n = 3) (n = 3) (n = 3) (n = 3) (n = 10) (n = 9) (n = 9)
Cmax (range), ng/mL 28,172 (16072–38,300) 28,829 (20072–38,300) 14,117 (3084–30,901) 80,873 (41225–124,800) 49,843 (3235–76,825) 55,187 (3451–100,440) 112,974 (59912–250,500) 87,182 (25197–171,780) 126,196 (53246–218,400)
AUC0-inf (range), ng·h/mL 32,004 (19865–43,524) 34,411 (21470–44,478) 22,343 (4587–52,228) 109,655 (58114–143,692) 90,439 (25777–135,436) 129561b(95030–164,092) 294,648 (157200–684,264) 261,255 (90854–417,087) 361,068 (109285–597,203)
Vz (range), mL/kg 66.3 (46.0–105) 58.7 (38.5–90.6) 499 (42.3–1083) 98.4 (55.9–121) 442 (42.8–1154) 44.2b(31.8–57.0) 76.6 (20.8–177) 95.5 (29.8–180) 92.2 (12.8–273)
CL (range), mL/h·kg 173 (115–252) 160 (112–233) 558 (95.7–1090) 107 (69.6–172) 184 (73.8–388) 83.1b(60.9–105) 84.9 (29.2–127) 101 (48.0–220) 73.5 (33.5–183)
Half-life (range), hours 0.261 (0.217–0.289) 0.250 (0.237–0.270) 0.507 (0.306–0.688) 0.701 (0.475–1.07) 1.20 (0.327–2.06) 0.367b(0.361–0.373) 0.723 (0.310–2.22) 0.715 (0.343–1.47) 0.767 (0.259–1.65)
  1. Abbreviations:
  2. AUC 0-inf area under the concentration-time curve from 0 h extrapolated to infinity, CL clearance, C max minimum concentration, LOPD late-onset Pompe disease, PK pharmacokinetics, Vz volume
  3. aLOPD subject cohorts received 5 mg/kg (n = 3), 10 mg/kg (n = 3), or 20 mg/kg (n = 10 at week 1; n = 9 at week 24) of reveglucosidase alfa as a weekly 2-h infusion for 24 weeks
  4. b n = 2 for this parameter