Pfizer Inc. and biopharmaceutical company, Protalix BioTherapeutics, Inc. announced that the US Food and Drug Administration (FDA) approved Elelyso (taliglucerase alfa) for injection for paediatric patients. Elelyso is therefore now indicated for long-term enzyme replacement therapy (ERT) for adult and paediatric patients with a confirmed diagnosis of type 1 Gaucher disease.
"The approval of Elelyso to treat paediatric patients with type 1 Gaucher disease provides physicians another treatment option for this rare and potentially debilitating disease,” said Rory O’Connor, senior vice president, global medical affairs, Global Innovative Pharma Business, Pfizer Inc. “This paediatric indication, along with the recent announcement that Elelyso received kosher certification by the Orthodox Union (OU), reinforces the ongoing commitment of Pfizer to addressing the needs of the Gaucher community.”
The safety and efficacy of Elelyso were assessed in fourteen pediatric patients with type 1 Gaucher disease in two clinical trials. The first trial consisted of nine patients in a 12-month, multi-center, double-blind, randomized study in treatment-naïve patients aged two to 13 years. At the end of the 12-month study, therapeutic efficacy of Elelyso was demonstrated, as measured by a decrease in spleen and liver volume and an increase in platelet count. A second trial consisted of 5 paediatric patients aged 6 to 16 years who were switched from imiglucerase to Elelyso. The trial was a 9-month, multi-center, open-label, single-arm study in patients who had been receiving treatment with imiglucerase at dosages ranging from 9.5 units/kg to 60 units/kg every other week for a minimum of 2 years. Elelyso was administered for 9 months at the same dose as each patient’s previous imiglucerase dose. If needed, adjustment of dosage was allowed during the study in order to maintain stability of clinical parameters. Mean spleen and liver volume, platelet count and hemoglobin value remained stable through 9 months of Elelyso treatment.
The recommended dosage of Elelyso 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.
Patients previously treated on a stable dosage of imiglucerase are recommended to begin treatment with Elelyso at that same dosage when they switch from imiglucerase to Elelyso. Dosage adjustments can be made based on achievement and maintenance of each patient’s therapeutic goals.
Serious hypersensitivity reactions, including anaphylaxis, have occurred in some patients treated with Elelyso. The most common adverse reactions for Elelyso in clinical trials were itching, flushing, headache, joint pain, pain in extremity, abdominal pain, vomiting, fatigue, back pain, dizziness, nausea and rash. Vomiting occurred more often in paediatric patients than adults.
“While type 1 Gaucher disease can manifest in childhood or adulthood, the disease more often presents during childhood,” said Paige Kaplan, MB, BCh, Section of Biochemical Genetics (Metabolic Diseases), Children's Hospital of Philadelphia. “It is important that children with this disease have access to a range of FDA-approved treatment options that are effective.”
As part of its ongoing commitment to helping address the unmet medical needs of people with rare diseases, Pfizer also offers a specialized support programme -- called Gaucher Personal Support (GPS) -- for people living with Gaucher disease. Pfizer GPS is a one-stop resource for personalized patient support and specialty pharmacy services for patients and healthcare professionals. Pfizer GPS employs a dedicated and caring team of healthcare specialists who are available to help patients with Gaucher disease and their caregivers with reimbursement assistance, coordinating and locating infusion services and ongoing pharmacy support.
Elelyso for injection is supplied as 200 units per vial and is available by prescription only.
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.
Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx.