Genzyme, a company Sanofi SA (ADR)(NYSE:SNY), announced that the Ministry of Health, Labor and Welfare (MHLW) has given marketing approval for Cerdelga® which is the only oral therapy for the treatment of adults who are suffering from Gaucher disease type 1 in Japan.
Cerdelga was approved by the U.S. Food and Drug Administration earlier in August 2014, by the European Commission in January 2015, and by the Australian Therapeutics Goods Administration in February 2015.
The the Ministry of Health, Labor and Welfare approval was based on data received from the clinical development program of Cerdelga which is the largest clinical research program that has ever been conducted in Gaucher disease type 1, the trial had approximately 400 patients who were treated in 29 countries.
3 Phase Clinical Trials:
The Cerdelga development program included three Phase 3 clinical trials. In ENGAGE, the Phase 3 treatment-naïve, placebo-controlled trial, improvements were observed after 9 months on Cerdelga in the spleen size, the platelet levels, liver volume and hemoglobin levels.
ENCORE, the second Phase 3 trial, was designed specifically for the assessment of disease stability in the patients who were previously treated with enzyme replacement therapy. That clinical trial met the pre-specified criteria for the non-inferiority to an enzyme replacement therapy (imiglucerase).
EDGE, the third Phase 3 trial, included 10 Japanese patients. It was designed for the evaluation of different dosing frequencies of Cerdelga in the patients who were previously treated with enzyme replacement therapy or treatment naïve Gaucher disease patients. The third phase trial included an interim analysis of the efficacy and safety in the Japanese patients. The patients in all three Phase 3 studies are still receiving Cerdelga in the extension periods and the majority of these patients are in their 4th or 5th year of treatment.
Richard Peters’ Statement:
The Acting Head of Rare Diseases at Genzyme, a company Sanofi SA (ADR)(NYSE:SNY), Richard Peters, MD, Ph.D. stated that the clinical development program conducted for Cerdelga represented a global effort throughout the Gaucher community, and the company was grateful for the efforts of Japan to provide access to Cerdelga to patients in their country. He added that the program demonstrated long-term commitment of Genzyme to this disease community, and the company recognizes the collaborative efforts of the physician and the patient communities as the drivers that have made this progress possible.