The FINANCIAL -- Pfizer and Protalix BioTherapeutics announced that the European Medicines Agency (EMA)’s Committee for
Medicinal Products for Human Use has adopted an Opinion
recommending against the Marketing Authorization of taliglucerase alfa,
an enzyme replacement therapy for the treatment of Gaucher
According to Pfizer Inc., as part of its Opinion, the CHMP gave a positive risk-benefit assessment for taliglucerase alfa concluding that the benefits of the medicine outweighed its risks in the treatment of Type 1 Gaucher disease.
Despite the positive risk-benefit assessment, the Committee could not recommend Marketing Authorization due to Shire’s velaglucerase alfa, which received prior Marketing Authorization with orphan drug designation for the same condition. Therefore, Shire’s treatment has orphan market exclusivity in the European Union for ten years from the time of its authorization in August 2010. Pfizer pursued a request for derogation from Shire’s orphan market exclusivity based on a number of factors. This request, however, was denied.
“While we are disappointed by the CHMP’s 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 Diem Nguyen, General Manager Biosimilars. “Pfizer will continue to work with relevant stakeholders to determine appropriate next steps.”
Pfizer and Protalix are dedicated to the treatment of Gaucher disease worldwide and continue to move forward with other global regulatory filings for taliglucerase alfa. Taliglucerase alfa was approved by the U.S. Food and Drug Administration on May 1, 2012 for the long term enzyme replacement therapy (ERT) of adults with a confirmed diagnosis of Type 1 Gaucher disease.
On November 30, 2009, Pfizer and Protalix BioTherapeutics, Inc. entered into an agreement to develop and commercialize taliglucerase alfa. Under the terms of the agreement, Pfizer received exclusive worldwide licensing rights for the commercialization of taliglucerase alfa, while Protalix retained the exclusive commercialization rights in Israel.