FDA Grants Approval for ELELYSO to treat Pediatric Patients with Type 1 Gaucher Disease

ELELYSO is the first FDA-approved plant cell-based recombinant therapeutic protein and is currently marketed in the United States by Pfizer Inc.
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WASHINGTON - Sept. 2, 2014 - PRLog -- Pfizer and Protalix BioTherapeutics, Inc received approval from the FDA for ELELYSO injectable therapy to treat Type 1 Gaucher disease in pediatric patients. The drug is indicated for long-term enzyme replacement therapy (ERT) for adult and pediatric patients 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.”

FDA approval stems from two clinical trials assessing 14 pediatric patients. The first trial involved nine patients aged two to thirteen years in a 12 month, multi-center, double-blind, randomized study. ELELYSO produced stellar clinical results as therapeutic efficacy demonstrated a decrease in spleen and liver volume as well as an increase in platelet count. The second trial assessed 5 pediatric patients aged 6 to 16 years  who were switched from imiglucerase to ELELYSO. ELELYSO was administered at dosages ranging from 9.5 units/kg to 60 units/kg every other week for a minimum of 2 years for 9 months in comparison to imiglucerase in a multi-center, open-label, single-arm study. According to the data, mean spleen and liver volume, platelet count and hemoglobin value remained stable through 9 months of ELELYSO treatment.

“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.” The recommended dosage is 60 units per kg of body weight  for treatment-naïve adult and pediatric patients four years of age and older as a 60 to 120 minute intravenous infusion

Gaucher is a rare inherited disease affecting fewer than 10,000 people worldwide. In patients affected, they do not have enough of an enzyme, β-glucosidase (glucocerebrosidase) to break down a certain type of fat molecule called glucocerebroside. As a result the fat molecule builds up in the cells, causing lipid engorged cells and large accumulation in organs and tissues, particularly the spleen, liver and bone marrow. Due to the high unmet medical need for people with rare diseases, in this case Gaucher, Pfizer has established a specialized support program – called Gaucher Personal Support for individuals living with Gaucher.

With the additional indication of ELELYSO to treat Type 1 Gaucher disease in pediatric patients, industry analysts expect a boost in sales further strengthening the companies’ portfolio primarily that of Protalix BioTherapeutix. With such industry leaders paving the way with new beneficial therapeutic breakthroughs, they will need both certified and qualified individuals to promote their diversified product portfolio. When industry leaders look for new candidates for sales/marketing positions, they look toward individuals that are industry trained. Companies are looking for people that have the background to sell their product both proficiently and efficiently.

The CNPR is a federally trademarked certification you can earn by demonstrating the necessary knowledge in pharmacology, medical terminology, physiology, and regulations for selling pharmaceuticals. The CNPR examination is dedicated to increasing the professional level of NAPSR members and to developing meaningful and ethical standards fully accepted by both its members and members of the pharmaceutical community.

The CNPR® Program represents a level of industry achievement and a demonstrated knowledge of pharmacology, medical terminology, pharmaceutical selling guidelines, physician selling techniques and industry standards. Pharmaceutical sales candidates who have this training will differentiate themselves from other individuals looking for open positions. Upon successful completion of the training each student will receive the Certification Mark of the CNPR®. CNPR® graduates should immediately utilize the NAPSRx® Career Center to apply for pharmaceutical sales positions.

Individuals that are interested in becoming a Certified National Pharmaceutical Representative (CNPR) can contact the National Association of Pharmaceutical Sales Representatives(NAPSRx) for more information or visit the website at http://napsronline.org/

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