Kyowa Kirin Co., Ltd.

The achondroplasia market across the seven major markets—the United States, Germany, France, Italy, Spain, the United Kingdom, and Japan—was valued at approximately USD 600 million in 2025 and is expected to grow at a compound annual growth rate of approximately 11% from 2026 to 2036, according to a new report from ResearchAndMarkets.com. The diagnosed prevalent population in these markets was estimated at approximately 28,500 cases in 2025, with the United States representing the largest patient population and commercial market at approximately 14,500 cases. The treatment landscape is shifting toward targeted therapies, with approved drugs VOXZOGO (vosoritide) and YUVIWEL (navepegritide; TransCon CNP) leading the way, while emerging pipeline candidates include infigratinib (BBP-831/BGJ398) from QED Therapeutics, a BridgeBio company, in collaboration with Novartis and Kyowa Kirin, RBM-007 (umedaptanib pegol) from RIBOMIC, and BMN 333 from BioMarin Pharmaceutical. Recent developments include RIBOMIC's Phase II summary and Phase III application in Japan, BridgeBio's positive Phase III PROPEL 3 results for infigratinib, and BioMarin's Phase I data for BMN 333. The report highlights opportunities in earlier genetic diagnosis, targeted FGFR3 therapies, and long-acting CNP analogs, while noting unmet needs such as limited treatment options across age groups and high treatment costs.

Cognizant and Benchling have been selected as partners to accelerate Kyowa Kirin's R&D by implementing Benchling's AI platform at the company's Tokyo Research Park and Fuji Research Park in Japan. The implementation aims to centralize data management, enhance molecular design processes, and automate workflows, enabling researchers to conduct experiment planning, execution, data collection, and AI use within a single environment. Kyowa Kirin expects the platform to automate experimental design and data collection, accelerating the research cycle from drug discovery target identification to new drug candidate selection. Cognizant will provide end-to-end support including platform setup, data migration, system implementation, and ongoing maintenance, while Benchling's platform connects directly with laboratory instruments to capture structured, searchable data automatically. The partnership is intended to improve researcher productivity, support informed decision-making, and establish a next-generation drug discovery foundation for Kyowa Kirin.

The U.S. Food and Drug Administration has selected Kyowa Kirin's Sanford manufacturing facility for the FDA PreCheck Pilot Program. The program enables early-stage regulatory engagement during the construction of the state-of-the-art biologics facility in Sanford, North Carolina, which broke ground in late 2024 and is expected to be operational by 2027. It is designed to optimize current Good Manufacturing Practices compliance and validation, accelerate commercial production timelines, and strengthen supply chain resilience for rare disease patients. Kyowa Kirin's Chief Supply Chain Officer Toshiyuki Kurata called the selection a transformative milestone, noting the facility is the company's first manufacturing footprint in the U.S. The PreCheck Program replaces sequential reviews with a proactive two-phase framework, including a facility readiness phase using a Type V Drug Master File for real-time FDA feedback and an application submission phase with structured pre-submission engagements to streamline Chemistry, Manufacturing, and Controls reviews.

Two indirect treatment comparison analyses presented at the World Congress of Cutaneous Lymphoma 2026 show that mogamulizumab is associated with improved overall survival compared to standard of care in patients with relapsed or refractory mycosis fungoides or Sézary syndrome. In the Australian study, median overall survival was not reached for mogamulizumab versus 31.0 months for vorinostat, with a hazard ratio of 0.48. In the Danish study, median overall survival was not reached for mogamulizumab versus 17.0 months for standard of care, with a hazard ratio of 0.38. The research, funded by Kyowa Kirin, used patient-level data from the MAVORIC trial and real-world registries to address evidence gaps where direct comparative survival data have been limited.

Eli Lilly and Regeneron Pharmaceuticals are among seven drug companies selected by the US FDA for its PreCheck Pilot Program, which aims to improve US drug manufacturing capabilities. The program offers earlier FDA engagement and a more predictable regulatory pathway for companies planning to manufacture drugs for the US market. Other selected companies include Amneal Pharmaceutical and Kyowa Kirin.

The XLH Network and Kyowa Kirin have launched the XLH Community Guidebook, a first-of-its-kind online resource for people living with X-linked hypophosphatemia, a rare genetic disease affecting bones and muscles. The Guidebook, available at www.XLHguidebook.com, was developed with input from patients, families, and experts to provide practical, evidence-based information tailored to each life stage from childhood through adulthood. It covers topics such as managing symptoms, coordinating care, and navigating transitions like school and work, and can be explored by topic or age group. While the initial launch is a joint effort, future editions will be managed solely by The XLH Network after the site transitions at the end of 2026.