PBI49 THE EXPEDITED REGULATORY PROGRAMS FOR REGENERATIVE MEDICINES IN US, EU AND JAPAN

Abstract

Several expedited regulatory pathways were established in different countries to accelerate the regenerative medicines (RMs) approval process. This study aims to describe the characteristics of RMs receiving Regenerative Medicine Advanced Therapy (RMAT) designation in United States (US), Priority Medicine (PRIME) designation in European Union (EU) and SAKIGAKE designation in Japan. RMs with PRIME designation and SAKIGAKE designation were extracted from European Medicine Agency (EMA) and Pharmaceuticals and Medical Devices Agency (PMDA) website, respectively. Press releases by pharmaceutical companies and Alliance for Regenerative Medicine (ARM) database were searched to identify RMs with RMAT designation. Up until May 2019, 28 RMs were granted RMAT designation in US, 23 were granted PRIME designation in EU and 11 were granted SAKIGAKE designation in Japan. Five products were granted 2 designations. Two RMs with PRIME designation were approved in EU, one RM with SAKIGAKE designation was approved in Japan, while no RM with RMAT designation was approved in US. Cell therapy (N=31, 50%) accounted for the largest proportion of RMs with above accelerated designations. Oncology disease (N=15, 24.19%) had the most number of RMs with designations, followed by neurology disease (N=12, 19.35%) and hematology disease (N=10, 16.13%). 22 (35.48%) out of 62 RMs received orphan drug designations. Five RMs have submitted MA application in at least one jurisdiction, and 17 RMs (27.42%) are currently being investigated in phase 3 clinical trials. RMs with RMAT designation, PRIME designation or SAKIGAKE designation could benefit from more extensive dialogues and interactions with regulators to obtain guidance on regulatory aspects including the clinical development plans. Thanks to the expedited programs, faster patient access to promising curative therapies could be expected in the near future.