Pfizer and Protalix BioTherapeutics announce approval of Type 1 Gaucher disease treatment


NEW YORK — Pfizer and Protalix BioTherapeutics last week announced that the Food and Drug Administration approved Elelyso (taliglucerase alfa) for injection for pediatric patients.  Elelyso is therefore now indicated for long-term enzyme replacement therapy for adult and pediatric patients with a confirmed diagnosis of Type 1 Gaucher disease.


"The approval of Elelyso to treat pediatric patients with Type 1 Gaucher disease provides physicians another treatment option for this rare and potentially debilitating disease,” stated Rory O’Connor, SVP global medical affairs, Global Innovative Pharma Business at Pfizer. “This pediatric indication, along with the recent announcement that Elelyso received kosher certification by the Orthodox Union, reinforces the ongoing commitment of Pfizer to addressing the needs of the Gaucher community.”


The safety and efficacy of Elelyso were assessed in 14 pediatric patients with Type 1 Gaucher disease in two clinical trials. The recommended dosage of Elelyso for treatment of pediatric patients four years of age and older is 60 units/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 pediatric patients than adults. 


“While Type 1 Gaucher disease can manifest in childhood or adulthood, the disease more often presents during childhood,” noted Paige Kaplan, of the Section of Biochemical Genetics (Metabolic Diseases) at 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 program — called Gaucher Personal Support — 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 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/vial and is available by prescription only. 


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