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Newly Completed NDA for Taliglucerase Alfa Submitted for FDA Review

CARMIEL, Israel--(BUSINESS WIRE)--Protalix Biotherapeutics, Inc. (NYSE-Amex: PLX) today announced the completion of its New Drug Application (NDA) submission with the U.S. Food and Drug Administration (FDA) for taliglucerase alfa, a plant-cell expressed form of glucocerebrosidase (GCD) for the potential treatment of Gaucher’s disease.

On December 1, 2009, Pfizer (NYSE: PFE - News) and Protalix entered into an agreement to develop and commercialize taliglucerase alfa for the treatment of Gaucher’s disease. The agreement gives Pfizer exclusive worldwide licensing rights to commercialize taliglucerase alfa while Protalix retains commercialization rights in Israel.

“With this submission, Protalix and Pfizer take a significant step forward in making a cost-effective treatment alternative available for Gaucher’s disease patients,” said Dr. David Aviezer, president and CEO of Protalix. “Our global plans include submitting additional regulatory applications for taliglucerase alfa in the near term.”

David Simmons, president and general manager of Pfizer’s Established Products Business Unit, stated, “Pfizer has assembled a team of experts with significant orphan and genetic disease experience dedicated to working with the worldwide Gaucher’s disease community. Upon the receipt of regulatory approvals, we are poised to quickly make taliglucerase alfa available to patients suffering with Gaucher’s disease who are in need of new treatment options.”

In addition, Protalix today announced the filing of its proposed pediatric investigation plan to the pediatric committee of the EMEA for a clinical study in patients between the ages of 2 and 18. This event triggers a milestone payment of $5 million by Pfizer to Protalix according to the agreement between the parties. The terms of the agreement calls for $55 million to be paid by Pfizer to Protalix in connection with certain regulatory milestones.

Taliglucerase alfa has been granted orphan product designation and fast track development status by FDA. Taliglucerase alfa is currently being provided to Gaucher’s patients in the U.S. under an expanded access protocol, as well as to patients in the European Union under a compassionate use protocol.

On December 2, 2009, Protalix held a medical meeting with the principal investigators involved with the Company’s taliglucerase alfa clinical trial program. At the event, the Company shared the full Phase III trial results that were submitted to FDA in the Company’s NDA filing. These data will be presented at the Annual Meeting of the Lysosomal Disease Network: WORLD Symposium 2010, February 10-12, in Miami, Florida.

About Gaucher’s disease
Gaucher’s disease, an inherited condition, is the most prevalent lysosomal storage disorder, with an incidence of about 1 in 20,000 live births. People with Gaucher’s disease do not have enough of an enzyme, â-glucosidase (glucocerebrosidase) that breaks down a certain type of fat molecule. As a result, lipid engorged cells (called Gaucher cells) amass in different parts of the body, primarily the spleen, liver and bone marrow. Accumulation of Gaucher cells may cause spleen and liver enlargement, anemia, excessive bleeding and bruising, bone disease and a number of other signs and symptoms.

Source: Protalix Biotherapeutics

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