|FDA accepts Protalix's resubmission|
|By Staff and Wire Reports|
|Wednesday, 17 August 2011 06:59|
The FDA deemed the resubmission a class 2, or 6-month, response and established February 1, 2012 as the Prescription Drug User Fee Act (PDUFA) date. Taliglucerase alfa is the Company's proprietary plant cell expressed recombinant form of human Glucocerebrosidase (GCD) which is being developed for the treatment of Gaucher disease.
Data from all twenty six adult patients enrolled in the Company's switchover trial of patients switched from Cerezyme® to taliglucerase alfa over the nine-month period, were included in the submission. The data supports the efficacy and safety data package showing that patients can be switched from imiglucerase (Cerezyme®) to taliglucerase alfa. One patient experienced a hypersensitivity reaction. The efficacy data demonstrates that mean hemoglobin and platelet count, spleen volume and liver volume remained stable. Patients enrolled in the trial were switched from imiglucerase (doses ranging from around 10-60 U/kg every other week) to an equivalent dose using the same number of units of taliglucerase alfa.
The submission also included data from treatment naive patients who completed the Company's pivotal Phase III trial and have continued to receive taliglucerase alfa for over 24 months in the Company's blinded long-term extension trial. These patients continued to show an improvement in efficacy and the drug was safe and well tolerated. Furthermore, those patients who were followed specifically for their bone parameters using Quantitative Chemical Shift Imaging (QCSI) MRI continued to show bone marrow improvement over time. Detailed data from the Company's switchover trial and long-term extension trial will be presented at upcoming medical meetings.Taliglucerase alfa is the Company's proprietary plant cell expressed recombinant form of human Glucocerebrosidase (GCD) which is being developed for the treatment of Gaucher disease.
"We look forward to working with the FDA in moving taliglucerase alfa through the regulatory review process over the next few months," stated Dr. David Aviezer, President and CEO of Protalix.
Regarding CMC, Protalix submitted further analyses and modifications of analyses previously submitted to the FDA to address their questions raised with regard to testing specifications and assay validation.
On November 30, 2009, Pfizer and Protalix BioTherapeutics, Inc. entered into an agreement to develop and commercialize taliglucerase alfa.
"Pfizer has worked closely with Protalix to achieve this important milestone," said Diem Nguyen, General Manager, Biosimilars. "Pfizer is ready to make taliglucerase alfa commercially available to Gaucher patients in the United States, if approved by the FDA."