FDA wants more data on Protalix drug [updated] Print E-mail
By Staff and Wire Reports   
Friday, 25 February 2011 08:43
On Friday morning, Protalix BioTherapeutics, Inc. (NYSE Amex: PLX; TASE: PLX), 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). 

“Importantly, it does not appear that the FDA has significant safety concerns with taliglucerase,” said Brian Abrahams, an analyst at Wells Fargo Securities in a note to clients today. “Rather, given the improving supply of Genzyme’s Cerezyme and availability of Shire’s velaglucerase, the agency likely prefers to wait for a more complete dataset for patients on taliglucerase prior to approval.”

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.

Protalix said FDA did not identify issues in its audit of clinical sites and did not ask for more clinical studies. The company said it will request a meeting as soon as possible to clarify the path to regulatory approval.  The company apparently has the data that the FDA is requesting, but regulators need clarification.

"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."

A meeting between the management teams of Protalix and Pfizer is scheduled so that they can plan a response strategy.

According to company officials, the firm has $35 million in cash- enough to carry them through for the next 12 months.

“We don’t think this is going to be a very long process,” Protalix Chief Executive Officer David Aviezer told analysts and investors on a conference call today. The company plans to work closely with Pfizer and request a meeting with the FDA as soon as possible to determine what needs to be done for approval.

Taliglucerase is the first product for Protalix. The medicine is designed to replace an enzyme missing in people with Gaucher disease, a disorder that causes fat to build up in the liver, spleen, bone marrow and nervous system.

Cerezyme had global sales of $719.6 million last year for Cambridge, Massachusetts-based Genzyme, a 42 percent decline from two years ago because of manufacturing issues. Vpriv, chemically known as velaglucerase alfa, generated sales of $143 million for Dublin-based Shire after being approved by the FDA in February 2010.


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