FDA approves Pfizer Elelyso to treat pediatric patients with type 1 Gaucher disease

August 28, 2014 / BIOON / -- Pfizer and protolix announced on August 28 that the FDA has approved elelyso (talilucerase alfa) injection for long-term treatment of pediatric patients with type 1 Gaucher disease This approval makes elelyso a long-term enzyme replacement therapy (ERT) that can be used in both type 1 adult and pediatric patients Elelyso's pediatric efficacy and safety were evaluated in two clinical trials involving 14 pediatric patients with type 1 Gaucher's disease One of the placebo-controlled studies involved nine patients, in which elelyso successfully stabilized the condition; the other involved five pediatric patients, who were successfully stabilized from enzyme replacement therapy Cerezyme to elelyso Previously, the FDA approved elelyso for use in adult patients with type 1 Gaucher's disease in May 2012 The drug is an enzyme replacement therapy, injected every two weeks Elelyso is also the first FDA approved drug based on plant cell expression system, which is produced by genetically engineered carrot cells This month, Sanofi's oral drug cerdelga (eliglutat) was approved by the FDA, which took 15 years to develop At present, enzyme replacement therapy (ERT) is the standard treatment for Gaucher's disease Enzyme replacement therapy (ERT) can degrade the fat deposits deposited in cells, while cerdelga can directly inhibit the accumulation of fat deposits in cells Analysts predict that cerdelga, as the first oral treatment drug, will completely overturn the current market pattern of injection-based drugs for Gaucher disease and compete with Pfizer's own Cerezyme, vriv of shire and elelyso of Pfizer For details, see "sharpening a sword in 15 years - cerdelga, an oral drug of Sanofi, has been approved by FDA - will overturn the market pattern of Gaucher disease" Gaucher disease is a rare genetic disease, which is caused by autosomal recessive inheritance The patients can not produce enough glucocerebrosidase (GCD) The deficiency of GCD results in the deposition of fatty substances in the spleen, liver, kidney and other organs The main signs are liver or spleen damage, anemia, low platelet and bone problems Currently, there are only 10000 patients with Gaucher's disease in the world, and the total number of patients in the United States is about 6000 Original English: Pfizer and protolix biotherapeutics announcement FDA approval of pediatric indication for elelyso Gamma (taliglucerase alfa) for Injection, for Intravenous Use for the Treatment of Type 1 Gaucher Disease Thursday, August 28, 2014 - 1:15pm EDT
Pfizer Inc (NYSE:PFE) and Protalix BioTherapeutics, Inc (NYSE-MKT:PLX, TASE:PLX) announced today that the U.S Food and Drug Administration (FDA) approved ELELYSO Gamma (taliglucerase alfa) for injection for pediatric patients ELELYSO is therefore now indicated for long-term enzyme replacement therapy (ERT) 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,” said Rory O’Connor, Senior Vice President, Global Medical Affairs, Global Innovative Pharma Business, Pfizer Inc “This pediatric 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 pediatric 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 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   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,” 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 program -- 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 Using Pfizer GPS, patients and healthcare professionals can access all support services 24/7 using one toll-free phone number, 1-855-ELELYSO (1-855-353-5976)   ELELYSO for injection is supplied as 200 units per vial and is available by prescription only   INDICATION ELELYSO Gamma is indicated for long-term enzyme replacement therapy (ERT) for adult and pediatric patients with a confirmed diagnosis of Type 1 Gaucher disease.