French drug maker Sanofi's (SNY) unit Genzyme announced that the Ministry of Health, Labor and Welfare (MHLW) of Japan has granted marketing approval for Cerdelga (eliglustat) as the only oral therapy for the treatment of adults with Gaucher disease type 1 in Japan.
The MHLW approval was based on data from the Cerdelga clinical development program, the largest clinical research program ever conducted in Gaucher disease type 1, with about 400 patients treated in 29 countries. The development program included three Phase 3 clinical trials.
Cerdelga, a novel glucosylceramide analog given orally, was designed to partially inhibit the enzyme glucosylceramide synthase, which results in reduced production of glucosylceramide, which is the substance that builds up in the cells and tissues of people with Gaucher disease.
Cerdelga was approved by the U.S. Food and Drug Administration in August 2014, the European Commission in January 2015, and the Australian Therapeutics Goods Administration in February 2015.
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