Japan's AMED names 8 projects for pre-designation orphan review

Japan's Agency for Medical Research and Development (AMED) named 8 projects for a pre-designation review as orphan drug commercialization candidates. These include three candidates from Tokyo-based Nobelpharma: N-acetylneuraminic acid for distal myopathy; NPC-14 (arbekacin sulfate) for Duchenne muscular dystrophy and a malaria vaccine. Candidates from other companies include additional indications of anticoagulant drugs from Tokyo-based Asahi Kasei Pharma; pustular psoriasis therapy from Tokyo-based SBI Biotech; GSK2315698 and GSK2398852 for systemic amyloidosis from GlaxoSmithKline ($GSK); therapeutic radiopharmaceuticals for malignant glioma from Tokyo-based Fujifilm RI Pharma; and achondroplasia therapy using anti-FGF2 aptamers from Tokyo-based Ribomic. AMED website


Using AI and RWD to Uncover Rare Disease Insights, Accelerate Commercialization and Improve Patient Outcomes

Wednesday, March 24 | 2pm ET / 11am PT

Learn how IPM.ai transformed real world data into real world insights to assist Audentes in their development of AT132 for the treatment of XLMTM. The session reviews how IPM.ia and Audentes collaborated to uncover the XLMTM patient population.