Long-term efficacy and safety results of taliglucerase alfa through 5years in adult treatment-naïve patients with Gaucher disease

Blood Cells Mol Dis. 2019 Sep:78:14-21. doi: 10.1016/j.bcmd.2016.07.002. Epub 2016 Jul 18.


Taliglucerase alfa, the first available plant cell-expressed recombinant therapeutic protein, is an enzyme replacement therapy approved for Gaucher disease (GD). PB-06-001, a pivotal phase 3, multicenter, randomized, double-blind, parallel-dose study investigated taliglucerase alfa 30 or 60U/kg every other week through 9months in treatment-naïve adults with GD; 30-month extension study PB-06-003 followed. Patients completing PB-06-001 and PB-06-003 could continue treatment in PB-06-007. Nineteen patients enrolled in PB-06-007 (30U/kg, n=8; 60U/kg, n=9; dose adjusted, n=2); 17 completed 5 total years of treatment. In these 3 groups, respectively, taliglucerase alfa resulted in mean decreases in spleen volume (-8.7, -6.9, -12.4 multiples of normal), liver volume (-0.6, -0.4, -0.5 multiples of normal), chitotriosidase activity (-83.1%, -93.4%, -87.9%), and chemokine (CC motif) ligand 18 concentration (-66.7%, -83.3%, -78.9%), as well as mean increases in hemoglobin concentration (+2.1, +2.1, +1.8mg/dL) and platelet count (+31,871, +106,800, +34,000/mm3). The most common adverse events were nasopharyngitis and arthralgia. Most adverse events were mild/moderate; no serious adverse events were considered treatment-related. These results demonstrate continued improvement of disease parameters during 5years of taliglucerase alfa therapy in 17 treatment-naive patients with no new safety concerns, extending the taliglucerase alfa clinical efficacy and safety dataset. This study was registered at www.clinicaltrials.gov as NCT01422187.

Keywords: Anemia; Chemokine (CC motif) ligand 18; Chitotriosidase; Enzyme replacement therapy; Gaucher disease; Hepatomegaly; Splenomegaly; Taliglucerase alfa; Thrombocytopenia.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Biomarkers
  • Enzyme Replacement Therapy* / methods
  • Female
  • Gaucher Disease / blood
  • Gaucher Disease / diagnosis
  • Gaucher Disease / drug therapy*
  • Glucosylceramidase / administration & dosage
  • Glucosylceramidase / adverse effects
  • Glucosylceramidase / therapeutic use*
  • Humans
  • Magnetic Resonance Imaging
  • Male
  • Middle Aged
  • Recombinant Proteins / administration & dosage
  • Recombinant Proteins / adverse effects
  • Recombinant Proteins / therapeutic use
  • Treatment Outcome


  • Biomarkers
  • Recombinant Proteins
  • Glucosylceramidase
  • taliglucerase alfa

Associated data

  • ClinicalTrials.gov/NCT01422187