New molecular entity: FDA approved taliglucerase alfa (Elelyso, Pfizer), a recombinant active form of the glucocerebrosidase enzyme for long-term enzyme replacement therapy (ERT) for adults with type 1 Gaucher disease.
On May 1, 2012, FDA approved taliglucerase alfa (Elelyso, Pfizer), a recombinant active form of the glucocerebrosidase enzyme for long-term enzyme replacement therapy (ERT) for adults with type 1 Gaucher disease.
Efficacy. The efficacy of taliglucerase alfa has been assessed as part of two, 9-month studies of adult patients with type 1 Gaucher disease. The first was in 31 patients naïve to ERT with enlarged spleens and thrombocytopenia (platelets <120,000/mm3 ) whom were randomly assigned to receive either 30 or 60 units/kg dosing. Both doses of taliglucerase alfa were found effective in reducing spleen volume from baseline (29% in patients receiving the 30 units/kg dose and 40% in patients receiving the 60 units/kg dose). Improvements in liver volume, blood platelet counts, and hemoglobin levels were also observed. In the second study, 25 patients with type 1 Gaucher disease who were receiving imiglucerase (another ERT) for at least 2 years prior where switched to taliglucerase alfa at the same dose. The study again showed taliglucerase alfa to be effective at maintaining spleen and liver volumes, blood platelet counts, and hemoglobin levels.
Safety. The most common adverse reactions observed during clinical studies were infusion reactions-occurring in 44% to 46% of patients-and including headache, chest pain or discomfort, asthenia, fatigue, urticaria, erythema, increased blood pressure, back pain, arthralgia, and flushing, and less commonly, angioedema, wheezing, dyspnea, coughing, cyanosis, and hypotension. Such infusion reactions could typically be managed by slowing the infusion rate, pre-treating with antihistamines, antipyretics and/or corticosteroids, and/or stopping and resuming treatment at a decreased infusion rate. Other commonly observed adverse reactions occurring in ≥10% of patients were upper respiratory tract infections/nasopharyngitis, pharyngitis/throat infection, headache, arthralgia, influenza/flu, urinary tract infection/pyelonephritis, back pain and extremity pain. Anaphylaxis has also been observed in some patients receiving taliglucerase alfa and is grounds for immediate drug discontinuation.