Endocrinology: Expanding the understanding of our drugs Audited

The aim of our Endocrinology research and development work is to offer patients with growth disorders and selected metabolic diseases new therapeutic options. We also want to continuously enhance the understanding of the products in our portfolio. In the PKU area we started the Phase IIIb trial SPARK in June. It is investigating the effects of Kuvan® in 50 European PKU patients younger than four years of age, for whom the drug is not yet approved. Additionally, with KAMPER, a multinational observational registry which started in 2009, we are studying the long-term safety of Kuvan®. Patient recruitment is still underway. For our growth hormone Saizen® we are using a two-pronged strategy in select countries to investigate how patients can achieve their full outcome potential. In the multinational observational study ECOS, which began in February, we are assessing patient adherence and its impact on treatment outcomes. The study aims to recruit and monitor at least 1,000 pediatric patients receiving Saizen® therapy via the Easypod™ injection device over a period of up to five years. In the pharmacogenetic program PREDICT, we are studying genetic markers that correlate with response to growth hormone treatment.