In August of 2014, the FDA approved ELELYSO for long-term enzyme replacement therapy (ERT)
for pediatric subjects with a confirmed diagnosis of Type 1 Gaucher disease. The recommended
dosage for treatment-naïve adult and pediatric subjects 4 years of age and older is 60 units
per kg of body weight administered every other week as a 60 to 120 minute intravenous
infusion. As a postmarketing commitment, the Sponsor agreed to evaluate the pharmacokinetics
(PK), pharmacodynamics (PD), and safety of Elelyso (taliglucerase alfa) in pediatric subjects
with Type 1 Gaucher Disease. in at least 5 subjects with body weight less than 15 kg; at
least 5 subjects with body weight 15 to less than 20 kg; and at least 5 subjects with body
weight of 20-25 kg with Type 1 Gaucher disease dosed at 60 units/kg every other week.
When applicable, PD measurements for children enrolled in the PK study may be obtained
through the taliglucerase alfa registry (PMR 1895-5) and will include organ volumes (spleen
and liver), hematological values (hemoglobin and platelets) as well as growth (height and
weight) data. Safety data, including any serious hypersensitivity reactions, such as
anaphylaxis, as well as changes in antibody status (ie, detection and titers of binding and
neutralizing antibodies, and detection of IgE antibodies), will also be collected through the
taliglucerase alfa registry.