ELELYSO

What it is

Elelyso contains taliglucerase alfa recombinant plant carrot (rpc). Taliglucerase alfa rpc is a recombinant form of human glucocerebrosidase expressed in genetically modified carrot plant cells in suspension that naturally bears terminal mannose structures for targeting macrophages. Elelyso is supplied as 200 units powder for injection, with each vial containing 200 units of taliglucerase alfa rpc.

Approved indications

— Long-term enzyme replacement therapy for adult and paediatric patients with a confirmed diagnosis of Type 1 Gaucher disease associated with splenomegaly, hepatomegaly, anaemia, or thrombocytopenia.

Dosing overview

Initial doses of Elelyso in adult and paediatric (2 years to 17 years of age) patients range from 30 units/kg to 60 units/kg of body weight once every 2 weeks, depending on the clinical assessment of the treating physician. Due to the heterogeneity and the multi-systemic nature of Gaucher disease, dosage must be individualised to each patient, and dose requirements may increase or decrease based on achievement of therapeutic goals, as assessed by regular comprehensive evaluations of the patient's clinical manifestations. After reconstitution and dilution, the total volume of prepared solution is administered by intravenous infusion over a period of 60 minutes to 120 minutes.

Key safety warnings

As with any intravenous protein product, infusion-related reactions and hypersensitivity reactions, including anaphylaxis are possible, and infusion-related reactions and allergic hypersensitivity reactions have been reported with Elelyso. Infusion-related reactions usually represent symptoms occurring within 24 hours of the infusion and are not necessarily linked to anaphylaxis or hypersensitivity, and may include symptoms such as arthralgia, headache, vomiting, flushing, pruritus, pain in extremity, diarrhoea, chest discomfort, feeling hot, muscle spasms, tremor and throat irritation. They can usually be managed successfully and patients can continue on therapy by slowing the infusion rate and/or stopping and resuming treatment with a decreased infusion rate. As with other therapeutic proteins, the development of immunoglobulin G (IgG) anti-drug antibodies (ADA) to taliglucerase alfa has been described, and hypersensitivity reactions occur in patients with and without ADA, but are more common among those with ADA some of whom experience anaphylactic reactions. Pulmonary hypertension is a known complication of Gaucher disease, patients with respiratory symptoms should be evaluated for the presence of pulmonary hypertension, and routine evaluation to detect the presence of pulmonary hypertension after diagnosis of Gaucher disease and over time is recommended. The occurrence of allergic reactions to Elelyso in patients with known carrot allergies is currently unknown and has not been studied in clinical trials; therefore, caution should be exercised in treating such patients.

Contraindications

Elelyso is contraindicated in severe allergic reactions to taliglucerase alfa rpc, any excipient components of the product, or other similar glucocerebrosidase enzymes.

Regulatory history

Elelyso was first approved on 21 May 2014. Elelyso taliglucerase alfa rpc 200 units powder for injection is registered on the ARTG (entry 207695) under licence category RE, with first listing on 21 May 2014.