Pfizer Inc. announced that researchers presented new data which expand on the existing body of data for Elelyso (taliglucerase alfa) for injection in pediatric patients with Type 1 Gaucher disease.  These data, which were released in an oral presentation at the 11th Annual WORLDSymposium in Orlando, include results from a phase 3, multi-center, extension trial evaluating the long-term efficacy and safety of Elelyso in pediatric patients with Type 1 Gaucher disease who were treatment-naïve or previously treated with imiglucerase.

This pediatric-specific extension trial included 15 patients aged two to 18 years from two study cohorts.  The first study cohort included 10 previously treatment-naïve pediatric patients treated with Elelyso (9 who completed) for up to 36 months (the first 24 months of which were conducted in a double-blind fashion) at 30 units per kg or 60 units per kg.  The second study cohort included five pediatric patients who were previously switched from imiglucerase (2 who completed) and administered Elelyso for a total of 33 months at the same dose as each patient’s previous imiglucerase dose.  At the end of the study, treatment-naïve patients treated with Elelyso for 36 months demonstrated clinical improvements, as measured by a decrease in spleen and liver volume and an increase in platelet count.  In patients previously switched from imiglucerase to Elelyso, mean spleen and liver volume, platelet count and hemoglobin value remained stable through 33 months of Elelyso treatment.

The most common adverse events reported were upper respiratory tract infection, cough, headache, abdominal pain, diarrhea, nasopharyngitis, pain in extremity, swollen or enlarged lymph nodes and fever.  

“The data presented at the WORLDSymposium contribute to our understanding of Elelyso as a long-term enzyme replacement therapy for patients with a confirmed diagnosis of Type 1 Gaucher disease,” said lead trial investigator Ari Zimran, MD, Gaucher Clinic, Sha’are Zedek Medical Center, Jerusalem, Israel.  “This study further underscores the clinical benefit of ongoing treatment with Elelyso in pediatric patients with Type 1 Gaucher disease.”

Elelyso is approved in the United States for long-term enzyme replacement therapy (ERT) for adult and pediatric patients with a confirmed diagnosis of Type 1 Gaucher disease, an inherited lysosomal storage disorder in humans that affects an estimated 10,000 people worldwide.  The recommended dosage of Elelsyo for treatment-naïve adult and pediatric patients four 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.

Elelyso (taliglucerase alfa) for injection is indicated for long-term enzyme replacement therapy (ERT) for adults and children with a confirmed diagnosis of Type 1 Gaucher disease.

Gaucher disease is an inherited lysosomal storage disorder in humans that affects an estimated 10,000 people worldwide and can cause severe and debilitating symptoms, including: enlargement of the liver and spleen, various forms of bone disease, easy bruising, and anemia (a low number of red blood cells).

Gaucher disease consists of varying degrees of severity; it has been sub-divided into three subtypes - Types 1, 2, and 3 - according to the presence or absence of neurological involvement. Type 1, the most common, is found at a higher frequency among individuals who are of Ashkenazi Jewish ancestry.

Rare diseases are among the most serious of all illnesses and impact millions of patients worldwide, representing an opportunity to apply our knowledge and expertise to help make a significant impact in addressing unmet medical needs.  The Pfizer focus on rare diseases builds on more than a decade of experience and a global portfolio of 22 medicines approved worldwide that treat rare diseases in the areas of hematology, neuroscience, inherited metabolic disorders, pulmonology, and oncology.