BUSINESS

Medipal/JCR’s MPS IIIB Therapy Snags Orphan Tag in US

May 8, 2025
Medipal Holdings and JCR Pharmaceuticals said on May 7 that the US FDA has granted orphan drug designation for JR-446, an investigational drug for mucopolysaccharidosis type IIIB (MPS IIIB), a lysosome disease also known as Sanfilippo syndrome type B.JR-446 is…

To read the full story

Related Article

BUSINESS

By Shunsuke Tani

Tariffs have been a defining feature of US trade policy under President Donald Trump’s second administration. Since Trump unveiled his “reciprocal” tariff policy in April 2025, the landscape has shifted repeatedly.In February 2026, the US Supreme Court invalidated tariffs imposed…

By Kohei Hori

As Japan’s budget season draws near, the Ministry of Finance (MOF) has renewed its push for a full-scale “off-year” drug…

By Ken Yoshino

Japan is pressing ahead with broader drug price cuts under its cost-effectiveness assessment (CEA) system, despite lingering questions over the…

By Kohei Hori

Members of a key reimbursement policy panel on October 28 raised no objections to expanding drug makers’ access to deliberations…

By Takashi Ebisawa

Eli Lilly’s breast cancer drug Imulrio (imlunestrant) is finally joining Japan’s NHI price list on October 14, nearly 300 days…

Japan’s Ministry of Health, Labor and Welfare (MHLW) on March 5 unveiled the revised NHI prices for drugs subject to…

Japan’s Central Social Insurance Medical Council (Chuikyo) on November 5 approved the reimbursement listing for a slate of new medicines,…

The Japanese government approved a bill to amend the Pharmaceuticals and Medical Devices (PMD) Act at its Cabinet meeting on…

Japan’s health ministry doled out regulatory approvals for a throng of new medicines on December 27 including Eli Lilly’s obesity…

Japan’s all-important reimbursement policy panel on December 20 approved an outline of drug pricing reforms for FY2024, which enshrines a…