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Elelyso (Taliglucerase alfa): A long-term enzyme replacement therapy for adults with type 1 Gaucher disease
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.
Coalition promotes important acetaminophen dosing reminders
November 18th 2014It may come as a surprise that each year Americans catch approximately 1 billion colds, and the Centers for Disease Control and Prevention estimates that as many as 20% get the flu. This cold and flu season, 7 in 10 patients will reach for an over-the-counter (OTC) medicine to treat their coughs, stuffy noses, and sniffles. It’s an important time of the year to remind patients to double check their medicine labels so they don’t double up on medicines containing acetaminophen.
Support consumer access to specialty medications through value-based insurance design
June 30th 2014The driving force behind consumer cost-sharing provisions for specialty medications is the acquisition cost and not clinical value. This appears to be true for almost all public and private health plans, says a new report from researchers at the University of Michigan Center for Value-Based Insurance Design (V-BID Center) and the National Pharmaceutical Council (NPC).
Management of antipsychotic medication polypharmacy
June 13th 2013Within our healthcare-driven society, the increase in the identification and diagnosis of mental illnesses has led to a proportional increase in the prescribing of psychotropic medications. The prevalence of mental illnesses and subsequent treatment approaches may employ monotherapy as first-line treatment, but in many cases the use of combination of therapy can occur, leading to polypharmacy.1 Polypharmacy can be defined in several ways but it generally recognized as the use of multiple medications by one patient and the most common definition is the concurrent use of five more medications. The presence of polyharmacy has the potential to contribute to non-compliance, drug-drug interactions, medication errors, adverse events, or poor quality of life.
Medical innovation improves outcomes
June 12th 2013I have been diagnosed with stage 4 cancer of the pancreas, a disease that’s long been considered not just incurable, but almost impossible to treat-a recalcitrant disease that some practitioners feel has given oncology a bad name. I was told my life would be measured in weeks.
