A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease

Adverse event(AE)=any untoward medical occurrence in clinical investigation participant administered a drug;it does not necessarily have to have causal relationship with this treatment. AE can therefore be any unfavorable&unintended sign (example,clinically significant abnormal laboratory value),symptom/disease temporally associated with use of drug whether or not it is considered related to drug. TEAE=any event emerging or manifesting at or after initiation of investigational product or any existing event that worsens in either intensity or frequency following exposure to investigational product. SAE=any untoward clinical manifestation of signs, symptoms, or outcomes(whether considered related to investigational product or not)&at any dose: results in death,is life-threatening,requires in-patient hospitalization/prolongation of hospitalization,results in persistent/significant disability/incapacity,results in congenital abnormality/birth defect,or is an important medical event.

An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory value), symptom, or disease temporally associated with the use of a drug whether or not it is considered related to the drug. A TEAE is defined as any event emerging or manifesting at or after the initiation of the investigational product or any existing event that worsens in either intensity or frequency following exposure to the investigational product.

An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory value), symptom, or disease temporally associated with the use of a drug whether or not it is considered related to the drug. An infusion-related AE was defined as an AE that 1) began either during or within 12 hours after the start of the infusion and 2) was judged as possibly or probably related to the study treatment.

Percentages were rounded off to the nearest single decimal place.

Clinical laboratory assessments included hematology and serum chemistry. Any clinically significant changes in the clinical laboratory assessment values based on the investigator's interpretation were reported. Only categories with at least one participant with event are presented.

Any abnormal changes in the urinalysis assessment values based on the investigator's interpretation were reported. Urinalysis comprised of the following parameters: bilirubin, ketones, glucose, nitrite, occult blood, protein, and urobilinogen.

Participants with atleast 1 abnormal change (above or below normal) in an infusion vital sign parameter of pulse, temperature, respiratory rate,& blood pressure were reported. Normal ranges for each vital sign parameter were, pulse (beats per minutes [bpm]): 40-100(≥12 years old), 55-95(≥6 but <12 years old), 65-110(≥2 but <6 years old); body temperature(degree Celsius[˚C]): 36.5 to 37.2(all age groups),respiration rate(breaths/minutes):12-24(≥12 years old),12-22 (≥6 but <12 years old),20-30(≥2 but <6 years old);systolic blood pressure(BP) [millimeters of mercury{mm Hg}]: high: ≥140 (≥18 years old), ≥20+ 80+ 2*age(<18 years old), low: <90 (≥18 years old), < -20+ 80+ 2*age(<18 years old); diastolic BP(mm Hg):high:≥90 (≥18 years old), ≥20+ (80+2*age)*(2/3) (<18 years old) low: <50 (≥18 years old), < -20+ (80+2*age)*(2/3) (<18 years old). As per planned analysis,data was collected in a combined manner for all participants irrespective of age. Only non-zero categories are presented.

Participants with clinically significant changes in any ECG measurement, such as PR, QRS, QT, corrected QT intervals, and heart rate based on the investigator's interpretation were reported.

Normalized liver volume was measured by abdominal magnetic resonance imaging (MRI) or computed tomography (CT) scan. Liver volume was normalized for percent body weight.

Spleen volume was normalized for percent body weight.

SF-36 Version 2 is a multipurpose, participant completed, short-form health survey with 36 questions that consists of an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures. Physical component summary (PCS) is mostly contributed by physical function (PF), role physical (RP), bodily pain (BP), and general health (GH). Mental component summary (MCS) is mostly contributed by mental health (MH), role emotional (RE), social function (SF), and vitality (VT). Each component on the SF-36 item health survey is scored from 0 (best) to 100 (worst). Total score ranges from 0-100 for each component summary (i.e., PCS and MCS), where higher scores are associated with less disability and better quality of life.

The CHQ-PF-50 is a 50-item questionnaire to be completed by the parents or guardians of children between 5 and 18 years of age. The 50 questions measure 14 domains (global health, physical functioning, role/social limitations (emotional/behavioral and physical), bodily pain/discomfort, behavior, global behavior, mental health, self-esteem, general health perceptions, change in health, parental impact (emotional and time), family activities, and family cohesion) which were summarized as the physical and psychological summary scores. Each summary score was transformed and could range from 0 to 100, with higher score indicating better physical and psychosocial health. A positive change from Baseline indicates improved well-being.

ng*min/mL denotes nanograms*minutes per milliliter.

mL/min/kg denotes milliliters per minutes per kilogram.