On Friday, Pfizer Inc. (NYSE:PFE) and Protalix Biotherapeutics Inc. (NYSE MKT:PLX; TASE: PLX) announced that the US Food and Drug Administration (FDA) had 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.
Protalix's share price rose by 8.3% on Wall Street on Friday. This follows prolonged weakness in the stock, which is down 24% over the past month.
"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 companies said that serious hypersensitivity reactions, including anaphylaxis, 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.”
Published by Globes [online], Israel business news - www.globes-online.com - on August 31, 2014
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