Graduate School of Design and Architecture, Nagoya City University, Nagoya 464-0083, Japan.
Ritsumeikan University, Osaka 567-8570, Japan.
Drug Discov Today. 2023 Oct;28(10):103755. doi: 10.1016/j.drudis.2023.103755. Epub 2023 Aug 28.
Rare disease (RD) patients face significant unmet therapeutic needs worldwide. However, orphan drugs approved in the United States, but not approved or developed in Japan, have increased rapidly with recently increasing US approvals, indicating greater RD drug loss in Japan. US/EU-based startups have become key players in RD drug R&D, significantly contributing to this drug loss trend. They successfully develop drugs in the United States by combining in-licensing with in-house drug discovery. Out-licensing to Japanese companies or large pharma is critical for expansion into Japan, with successes attributed to drug innovation, target indications, and transactional capabilities. These findings highlight the need to foster partnerships with startups and cultivate an ecosystem in Japan that nurtures local startups, to address drug loss and ensure access to promising drugs.
罕见病（RD）患者在全球范围内面临着巨大的未满足的治疗需求。然而，近年来，随着美国批准的孤儿药数量不断增加，虽然已经获得美国批准，但在日本尚未获得批准或开发的孤儿药数量也迅速增加，这表明日本的孤儿药损失更大。以美国和欧盟为基地的创业公司已经成为 RD 药物研发的关键参与者，这一趋势导致了 RD 药物的大量损失。这些公司通过引进许可和内部药物发现相结合，成功地在美国开发药物。对于拓展日本市场，将药物授权给日本公司或大型制药公司至关重要，成功的关键在于药物创新、目标适应症和交易能力。这些发现强调了日本需要与创业公司建立伙伴关系，培育一个有利于本地创业公司的生态系统，以解决药物损失问题，确保获得有前途的药物。
