Japan’s cost-effectiveness assessment (CEA) panel has approved a revised handling policy for the CEA system for FY2026, with the health…
BUSINESS
JCR’s Hurler Syndrome Drug Gets Orphan Status in US
JCR Pharmaceuticals said on February 12 that it has earned orphan drug designation for JR-171, a blood-brain-barrier (BBB)-penetrating α-L-iduronidase, for the treatment of mucopolysaccharidosis type 1 (Hurler, Hurler-Scheie, and Scheie syndrome).The company is running a PI/II trial globally for JR-171,…
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