EMA Recommends Against Marketing Authorization to Pfizer and Protalix for Gaucher Treatment

   By Saabira Chaudhuri 
 

The European Medicines Agency has recommended against marketing authorization for Pfizer Inc. (PFE) and Protalix BioTherapeutics Inc. (PLX) for their Gaucher disease treatment, taliglucerase alfa.

Protalix's shares were down 12% premarket to $5.83. The stock is up 34% year-to-date. Pfizer's shares were flat premarket at $22.60. The stock is up 4% year-to-date.

The European agency made a positive risk-benefit assessment, concluding that the benefits of the treatment outweighed its risks, but didn't recommend marketing authorization because Shire PLC's (SHPGY, SHP.LN) velaglucerase alfa has received prior marketing authorization with an orphan drug designation for the same condition. Shire's treatment has marketing exclusivity in the European Union for 10 years from August 2010, the time of authorization.

"While we are disappointed by the CHMP's [EMA's Committee for Medicinal Products for Human Use] recommendation, we are encouraged that the committee gave a positive risk-benefit assessment. The recommendation was based solely on orphan market exclusivity and not the safety and efficacy profile of taliglucerase alfa," said General Manager Diem Nguyen of Pfizer's Biosimilars division. "Pfizer will continue to work with relevant stakeholders to determine appropriate next steps."

Pfizer and Protalix BioTherapeutics entered into an agreement to develop and commercialize taliglucerase alfa, an enzyme replacement therapy, in November 2009. The treatment was approved by the Food and Drug Administration last month for the long term enzyme replacement therapy of adults with a confirmed diagnosis of Type 1 Gaucher disease.

According to the National Institute of Health's National Institute of Neurological Disorders and Stroke, Gaucher disease is the most common of the inherited metabolic disorder known as lipid storage diseases. Lipids are fatty materials that include oils, fatty acids, waxes, and steroids [such as cholesterol and estrogen]. Gaucher disease is caused by a deficiency of the enzyme glucocerebrosidase. Fatty materials can accumulate in the spleen, liver, lungs, bone marrow, and brain. Symptoms may include skeletal disorders, enlarged spleen and liver, liver malfunction, anemia, and yellow spots in the eyes.

Write to Saabira Chaudhuri at [e-mail]