Vidaza recommended in Japan for MDS
This article was originally published in Scrip
Nippon Shinyaku's Vidaza (azacitidine; NS-17) has received a positive approval opinion in Japan for the treatment of myelodysplastic syndromes (MDS).
The product, licensed from Celgene, was filed for the orphan indication in December 2009 and received a priority regulatory review. The submission contained data from a local pivotal clinical trial and other international studies, which have shown a survival benefit of around nine months for Vidaza compared with conventional treatments.
The recommendation, from an advisory committee of the ministry of health, labour and welfare, is for a disorder that affects an estimated 9,000 people a year in Japan. MDS is currently treated mainly through supportive care, although low-dose cytarabine or chemotherapy may also be used.
The condition is characterised by pre-leukaemic changes in the bone marrow that affect blood cell production, leading to fatigue, infections and fever, and it progresses to acute myelogenous leukaemia in around 40% of cases.
Azacitidine, an analogue of the DNA nucleoside cytidine, acts as a demethylating agent to promote the normalisation of blood cell production and also has a cytotoxic effect on abnormal bone marrow haematopoietic cells, but without major suppression of DNA synthesis.
It was approved as Vidaza in the US in 2004 and is marketed in around 20 countries. The UK health technology appraisal body NICE is re-examining its decision earlier this year not to recommend the drug for MDS and several types of leukaemia, following complaints from a number of patient groups (scripintelligence.com, 29 July 2010).
Under the exclusive Japanese licensing deal with Nippon Shinyaku, Celgene will receive undisclosed regulatory and sales milestone payments, plus royalties on sales.
vaccine recommendation
Also recommended for approval by the review committee was an attenuated combined measles and rubella vaccine produced by Japan's Kitasato Institute. Several other similar combined vaccines are already on the market, including one distributed by Takeda.