Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase.
Taliglucerase alfa is the first available plant cell-expressed human recombinant therapeutic protein. It is indicated for treatment of patients with type 1 Gaucher disease (GD) in adult and pediatric patients in several countries. Study PB-06-002 examined the safety and efficacy of taliglucerase alfa for 9 months in patients who previously received imiglucerase. The results of adult patients from Study PB-06-002 who continued receiving taliglucerase alfa in extension Study PB-06-003 for up to 36 months are reported here. Eighteen patients received at least one dose of taliglucerase alfa in Study PB-06-003; 10 patients completed 36 total months of therapy, and four patients who transitioned to commercial drug completed 30-33 months of treatment. In patients who completed 36 total months of treatment, mean percent (±standard error) changes from baseline/time of switch to taliglucerase alfa to 36 months were as follows: hemoglobin concentration, -1.0% (±1.9%; n = 10); platelet count, +9.3% (±9.8%; n = 10); spleen volume measured in multiples of normal (MN), -19.8% (±9.9%; n = 7); liver volume measured in MN, +0.9% (±5.4%; n = 8); chitotriosidase activity, -51.5% (±8.1%; n = 10); and CCL18 concentration, -36.5 (±8.0%; n = 10). Four patients developed antidrug antibodies, including one with evidence of neutralizing activity in vitro. All treatment-related adverse events were mild or moderate and transient. The 36-month results of switching from imiglucerase to taliglucerase alfa treatment in adults with GD provide further data on the clinical safety and efficacy of taliglucerase alfa beyond the initial 9 months of the original study. www.clinicaltrials.gov identifier NCT00705939. Am. J. Hematol. 91:661-665, 2016. © 2016 Wiley Periodicals, Inc.
National Centre for Inherited Metabolic Disorders, Mater Misericordiae University Hospital, Dublin, Ireland.
Gregory M
GM
Pastores
Department of Genetics, Emory University School of Medicine, Atlanta, Georgia.|Department of Ophthalmology, Emory University School of Medicine, Atlanta, Georgia.
Suma P
SP
Shankar
Clinical Center of Serbia, Clinic of Endocrinology, Diabetes and Metabolic Diseases, Belgrade University Medical School, Belgrade, Serbia.
Milan
M
Petakov
CIBERER, Hospital Universitario Miguel Servet, Zaragoza, Spain.
Pilar
P
Giraldo
Rambam Medical Center, Haifa, Israel.
Hanna
H
Rosenbaum
Mount Sinai Hospital, Toronto, Ontario, Canada.
Dominick J
DJ
Amato
Royal Melbourne Hospital, Victoria, Australia.
Jeffrey
J
Szer
Protalix BioTherapeutics, Carmiel, Israel.
Raul
R
Chertkoff
Protalix BioTherapeutics, Carmiel, Israel.
Einat
E
Brill-Almon
Gaucher Clinic, Shaare Zedek Medical Center, Jerusalem, Israel.
Ari
A
Zimran
D000328:Adult; D000368:Aged; D019742:Chemokines, CC / Q000187:drug effects; D057915:Drug Substitution; D056947:Enzyme Replacement Therapy / Q000379:methods*; D005260:Female; D005776:Gaucher Disease / Q000188:drug therapy*; D005962:Glucosylceramidase / Q000008:administration & dosage* / Q000627:therapeutic use; D006454:Hemoglobins / Q000032:analysis; D006596:Hexosaminidases / Q000187:drug effects / Q000378:metabolism; D006801:Humans; D008099:Liver / Q000473:pathology; D008297:Male; D008875:Middle Aged; D009929:Organ Size / Q000187:drug effects; D010976:Platelet Count; D013154:Spleen / Q000473:pathology; D016896:Treatment Outcome; D055815:Young Adult
C111792:CCL18 protein, human; D019742:Chemokines, CC; D006454:Hemoglobins; D006596:Hexosaminidases; C086400:chitotriosidase; D005962:Glucosylceramidase; C582473:taliglucerase alfa; C090568:imiglucerase