Study to Determine if Japanese New-biopharmaceuticals were Approved by FDA and EMA Authorities

抄録

Objective: To study if Japanese new-biopharmaceuticals (1985-2016), including nine hormones and cytokines, three enzymes, one other medicine, and three antibody medicines originating in Japan, were approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) authorities. Results and Discussion: Of the nine hormones and cytokines, the Ministry of Health and Welfare, Japan (MHW) approved the use of Mecasermin on October 5, 1994 and the FDA approved its use on August 30, 2005. Mecasermin is given for the long-term treatment of growth failure in children with severe primary insulin-like growth factor (IGF) deficiency or with growth hormone (GH) gene deletion. None of the three enzymes and one other pharmaceutical were approved. The three antibody medicines were approved by the Ministry of Health, Labour and Welfare, Japan (MHLW) / the Pharmaceuticals and Medical Devices Agency (PMDA) as follows: The EMA approved the use of Tocilizumab as a rheumatoid on January 16, 2009, and the FDA approved use on August 1, 2010. The FDA approved the use of Mogamulizumab for the treatment of relapsed or refractory mycosis fungoides (MF) or Sezary syndrome (SS) on August 8, 2018. The FDA approved the use of Nivolumab for the treatment of melanoma on December 22, 2014 and for the treatment of metastatic squamous non-small cell lung cancer on March 4, 2015. The EMA approved the use of Nivolumab for four types of treatment on June 19, 2015. In this article, I analyzed why the 12 other biopharmaceuticals were not approved by the FDA and/or EMA. The importance of the ICH guidelines for adapting new GCP(E6) in 1998, and that of the quality of biotechnological/biological products are discussed. The establishment of the PMDA in 2004, and history of enhancing the transparency of the review process are also discussed.