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 saabira.chaudhuri@dowjones.com