The Burrill Report


The U.S. Food and Drug Administration notified Protalix BioTherapeutics that it would not approved taliglucerase alfa, its experimental treatment for Gaucher disease, without additional information. Taliglucerase alfa is a form of glucocerebrosidase expressed by plant cells. The company said the main questions raised by the FDA relate to clinical and chemistry issues, manufacturing, and controls. The agency did not request additional clinical studies. In November 2009, Pfizer and Protalix entered into an agreement to develop and commercialize taliglucerase alfa.

Takeda Pharmaceutical said it voluntarily recalled formulations of Dasen, its anti-inflammatory enzyme preparation sold in Japan. The decision followed studies that found no statistical significance between Dasen and a placebo. Based on the results of the studies, the Committee on Reevaluation of the Pharmaceutical Affairs and Food Sanitation Council of the Ministry of Health, Labour and Welfare of Japan held a meeting in January to discuss whether to require future reevaluation of the the medicine with additional studies. Takeda studied the feasibility of conducting such studies. But even as it it determined Dasen’s efficacy would be confirmed through additional clinical trials with a revised study design, however, it has concluded that it would be difficult to conduct such studies. Takeda has been marketing Dasen in Japan since 1968 and the product has been prescribed by physicians to a number of patients for over 40 years. Dasen had sales of $82 million (Yen 6.7 billion). The recall is expected to have minimal impact on 2010 earnings.

The U.S. Food and Drug Administration warned Sanofi-Aventis for failing to report in a timely manner adverse reactions in people who used its drugs, Reuters reported. The agency said in a letter to Sanofi that it previously promised to take corrective steps to address deficiencies in its reporting. “We remain concerned that your .... adverse drug experience reporting system has not been fully validated, and may have resulted in inaccurate assessment and untimely submission of 15-day alerts,” the FDA said.

Salix Pharmaceuticals said it expects to get formal notification from the U.S. Food and Drug Administration that the agency will not approve a label expansion for Xifaxan that would include treatment of non-constipation irritable bowel syndrome. The company says the FDA would like to review retreatment information before it will consider the application for approval. The company said it will consider next steps once it gets formal notification from the agency. Xifaxan is currently approved for reduction in risk of overt hepatic encephalopathy recurrence in patients at least 18 years of age.

Switzerland-based Carbogen Amcis, a pharmaceutical process development and active pharmaceutical ingredient manufacturer, said it will eliminate 60 jobs, nearly 20 percent of its staff, as part of a restructuring to boost profitability. The company blamed the strong Swiss franc and the delayed effects of the financial crisis for necessitating the move.



February 25, 2011
http://www.burrillreport.com/article-fda_not_ready_to_approve_protalix%e2%80%99s_gaucher_disease_drug.html