|FDA Approves ELELYSO(TM); Auxilium To Present CCH Data For AUX-CC-867 MULTICORD|
|By Staff and Wire Reports|
|Thursday, 28 August 2014 17:54|
Pfizer Inc. (NYSE: PFE) and Protalix BioTherapeutics, Inc. (NYSE: PLX) announced the 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.
"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).
Auxilium Pharmaceuticals, Inc. (NASDAQ: AUXL), a specialty biopharmaceutical company, today announced that data from trials evaluating the use of collagenase clostridium histolyticum (CCH) for treating two Dupuytren's contracture (DC) cords concurrently in adult patients with a palpable cord will be presented at the upcoming 69th Annual Meeting of the American Society for Surgery of the Hand (ASSH) being held in Boston on September 18-20, 2014. XIAFLEX®(CCH) is a biologic approved in the U.S., EU, Canada and Australia for the treatment of adult DC patients with a palpable cord.
DC is a progressive hand disease that can present with multiple collagen "cords" that limit finger movement and hand function. It is estimated that 35 to 40 percent of annual surgical procedures in the U.S. are performed to treat more than one DC cord at a time1.
The AUX-CC-867 MULTICORD (MULtiple Treatment Investigation of Collagenase Optimizing the Resolution of Dupuytrens) study data examining the effect of delayed finger extension on patients who received two concurrent injections of CCH to treat two affected joints in the same hand were selected for the meeting's Best Paper Session. Additional findings from the MULTICORD study featured in meeting presentations include the safety and efficacy of CCH as a treatment for two affected joints in the same hand concurrently and an analysis of efficacy based upon baseline joint severity. Additionally, a podcast has been selected for presentation that describes an analysis of the MULTICORD study treating two affected joints in the same hand concurrently using local anesthesia prior to finger extension. Finally, data will also be presented from the AUX-CC-862 retreatment study examining the retreatment of recurrent contractures in DC joints that were previously treated with CCH.
"The data presented at this year's Annual ASSH Meeting help support the efficacy and safety profile of CCH as a non-surgical treatment option for treating two cords concurrently during one office procedure, expanded flexibility with timing of the finger extension procedure and the retreatment of recurrent joints in those who were previously treated with CCH," said Adrian Adams, Chief Executive Officer and President of Auxilium Pharmaceuticals.
Data to be presented on CCH include:
--Effect of Delayed Finger Extension on the Efficacy and Safety of Collagenase Clostridium Histolyticum Treatment for Dupuytren's Contracture(Clinical Paper 01, Selected for the Best Paper Session, Podium Presentation, Thursday, September 18, 2:00 p.m. ET)
--Prospective Multicenter, Multinational Study to Evaluate the Safety and Efficacy of Concurrent Collagenase Clostridium Histolyticum Injections to Concurrently Treat Two Dupuytren's Contractures in the Same Hand(Clinical Paper 47, Podium Presentation, Friday, September 19, 2:00 p.m. ET)
--Treatment of Recurrent Dupuytren's Contractures in Joints Previously Effectively Treated With Collagenase Clostridium Histolyticum: Final Results(Clinical Paper 48, Podium Presentation, Friday, September 19, 2:00 p.m. ET)
--Effect of Baseline Severity on the Safety and Efficacy of Concurrent Collagenase Clostridium Histolyticum Injections to Treat Two Dupuytren's Contractures(Clinical Paper 44, Podium Presentation, Friday, September 19, 2:00 p.m. ET)
--Use of Local Anesthesia During Finger Extension: Effects on Efficacy and Safety of Collagenase Clostridium Histolyticum for Concurrent Treatment of Two Dupuytren's Contractures( Podcast 1384)
The U.S. Food and Drug Administration is reviewing the Company's submission of a supplemental Biologics License Application (sBLA) requesting approval of XIAFLEX for the treatment of two Dupuytren's cords concurrently. The PDUFA date for the sBLA is October 20, 2014.
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Protalix BioTherapeutics, Inc. (NYSE MKT:PLX) (TASE:PLX), announced today that it will host a conference call on Wednesday, September 3, 2014 at 8:30am ET to discuss the recent approval of ELELYSO for pediatric patients as jointly announced with Pfizer on August 28, 2014.
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