News Column

Pfizer and Protalix BioTherapeutics Announce FDA Approval of Pediatric Indication for ELELYSO™ taliglucerase alfa for Injection, for Intravenous Use...

September 10, 2014



Pfizer and Protalix BioTherapeutics Announce FDA Approval of Pediatric Indication for ELELYSO™ taliglucerase alfa for Injection, for Intravenous Use for the Treatment of Type 1 Gaucher Disease

By a News Reporter-Staff News Editor at Biotech Week -- Pfizer Inc. (NYSE:PFE) and Protalix BioTherapeutics, Inc. (NYSE-MKT:PLX, TASE:PLX) announced that the U.S. Food and Drug Administration (FDA) approved ELELYSO™ (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 (see also Biotechnology Companies).

"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-naive 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-naive 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.

For more information about Gaucher disease, please visit www.ELELYSO.com. INDICATION ELELYSO™ is indicated for long-term enzyme replacement therapy (ERT) for adult and pediatric patients with a confirmed diagnosis of Type 1 Gaucher disease. IMPORTANT SAFETY INFORMATION Serious hypersensitivity reactions including anaphylaxis have occurred in some patients treated with ELELYSO (taliglucerase alfa) for injection, for intravenous use. When treated with ELELYSO your doctor should monitor you before and after infusion for reactions.

Medical support should be readily available when ELELYSO is given. Discontinue ELELYSO immediately if you show signs or symptoms of anaphylaxis during infusion and get immediate medical care. Signs and symptoms of anaphylaxis included hives, low blood pressure, flushing, wheezing, chest tightness, nausea, vomiting and dizziness.

Signs and symptoms of hypersensitivity included itching, swelling under the skin, flushing, redness, rash, nausea, vomiting, cough, chest tightness and throat irritation. These reactions occurred up to 3 hours after the start of infusion.

Management of hypersensitivity reactions is based on the severity of the reaction. Your doctor may manage the reactions by slowing or temporarily stopping the infusion, and/or treating with medications such as an antihistamine, a fever reducer and/or corticosteroids for mild reactions. Treatment with antihistamines and/or corticosteroids prior to infusion with ELELYSO may prevent these reactions from reoccurring. If severe hypersensitivity reactions occur, immediately stop the infusion of ELELYSO and get immediate medical care.

You should be carefully re-evaluated for treatment with ELELYSO if serious or hypersensitivity reactions including anaphylaxis occur.

The most common adverse reactions for ELELYSO are itching, flushing, headache, joint pain, pain in extremity, abdominal pain, vomiting, fatigue, back pain, dizziness, nausea and rash. Vomiting occurred more often in children than adults.

The recommended dosage of ELELYSO for adults and children who are 4 years of age and older and not taking another ERT is 60 units per kg of body weight given every other week as a 60 to 120 minute intravenous infusion.

As with all therapeutic proteins, including (enzyme replacement therapy) ERTs, there is a possibility of developing antibodies to ELELYSO. The relationship between developing antibodies and hypersensitivity reactions is not clear. Your doctor should monitor you for antibodies to ELELYSO if you have developed antibodies or if you have experienced hypersensitivity reactions to ELELYSO or other ERTs.

If you are pregnant, or plan to become pregnant, you should talk to your doctor about potential benefits and risks.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

For full prescribing information click here.

The health information contained herein is provided for educational purposes only and is not intended to replace discussions with a health care provider. All decisions regarding patient care must be made with a health care provider, considering the unique characteristics of the patient. This product information is intended only for residents of the United States. About 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).

Keywords for this news article include: Biotechnology Companies, Clinical Trials and Studies, Drugs, FDA Actions, Gastroenterology, Genetics, Government Agencies Offices and Entities, Hepatology, Intravenous Infusions, Nausea, Parenteral Infusions, Pediatrics, Pfizer Inc., Pharmaceutical Companies, Therapeutics, Therapy.

Our reports deliver fact-based news of research and discoveries from around the world. Copyright 2014, NewsRx LLC


For more stories covering the world of technology, please see HispanicBusiness' Tech Channel



Source: Biotech Week


Story Tools






HispanicBusiness.com Facebook Linkedin Twitter RSS Feed Email Alerts & Newsletters