MHLW JAPAN | CHUIKYO DRUG PRICING | August 13, 2026Source: Adapted and translated from Ministry of Health, Labour and Welfare (MHLW) / Chuikyo drug pricing documentatio

MHLW JAPAN | CHUIKYO DRUG PRICING | August 13, 2026Source: Adapted and translated from Ministry of Health, Labour and Welfare (MHLW) / Chuikyo drug pricing documentatio

MHLW JAPAN | CHUIKYO DRUG PRICING | August 13, 2026Source: Adapted and translated from Ministry of Health, Labour and Welfare (MHLW) / Chuikyo drug pricing documentatio

Drug Pharmacologic class Indication More information Cardiovascular Disease Juxtapid (lomitapide) Microsomal triglyceride transfer protein inhibitor

Recent rare disease developments include regulatory progress, new orphan drug designations, and treatment access updates across multiple regions. Highlights include FDA expansion of JUXTAPID for pediatric homozygous familial hypercholesterolemia, advancement of Sangamo’s ST-920 gene therapy BLA submission for Fabry disease, and a new every-four-weeks dosing option for Elfabrio in the European Union. Additional updates include orphan designations for therapies targeting Behçet’s disease, IgG4-related disease, palmoplantar pustulosis, and transplant rejection, alongside a Canadian approval for selumetinib in adults with neurofibromatosis type 1 and plexiform neurofibromas.