Elelyso

Treatment for Gaucher Disease

Update: Elelyso (taliglucerase alfa) Now FDA Approved - May 1, 2012

FDA Denies Taliglucerase Alfa New Drug Application

February 25, 2011 - On Friday morning, Protalix BioTherapeutics, Inc., announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) regarding the Company's New Drug Application (NDA) for taliglucerase alfa for the treatment of Gaucher disease. Some of biotech's most risk-averse firms and analysts had predicted that the FDA would approve the treatment candidate, but the FDA has raised questions about the NDA related to the clinical and chemistry, manufacturing and controls (CMC).

According to the company, in the clinical section, the FDA requested additional data from the Company's switchover trial and long-term extension trial. At the time the NDA was submitted, full data from these trials was not available. In the CMC section, the FDA requested information regarding testing specifications and assay validation.

"While we are disappointed by the receipt of the Complete Response Letter, we appreciate the FDA's efforts to complete the review of our NDA. We noted that the FDA did not request additional clinical studies. Moreover, the FDA inspected our manufacturing facilities finding them acceptable. FDA also did not identify any issues in its audit of our clinical sites," said Dr. David Aviezer, the Company's President and Chief Executive Officer. "Protalix will work with the FDA to determine next steps."

On November 30, 2009, Pfizer and Protalix BioTherapeutics, Inc. entered into an agreement to develop and commercialize taliglucerase alfa.

"Pfizer remains dedicated to the Gaucher community worldwide," said David Simmons, President and General Manager, Emerging Markets and Established Products Business Units, Pfizer Inc. "We will work closely with Protalix to address the requests from the FDA in a timely manner by providing technical, analytical and regulatory expertise."

Protalix will request a meeting with the FDA as soon as possible to clarify the path to regulatory approval.

2010 BioMed Reports All rights reserved.

Posted: February 2011

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