Date: 2015-02-12
Type of information: Presentation of results at a congress
phase: pediatric
Announcement: presentation of results at the 11th Annual Lysosomal Disease Network WORLD Symposium in Orlando
Company: Pfizer (USA - NY)
Product: Elelyso® (taliglucerase alfa)
Action
mechanism: enzyme replacement therapy. Elelyso® is the first FDA-approved plant cell-based enzyme replacement therapy for Gaucher disease.
Disease: Type 1 Gaucher Disease
Therapeutic area: Rare diseases - Genetic diseases
Country:
Trial details:
Latest
news: * On February 12, 2015, Pfizer 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.
