Bio News
Ultragenyx has priced Pabuvy, a gene therapy for Sanfilippo syndrome type A, at $3.95 million. The US Food and Drug Administration (FDA) approved the treatment as the first gene therapy for the disease, and Ultragenyx has launched its second gene therapy since August.
Along with the two approvals, Ultragenyx secured a priority review voucher that can be sold and signaled cost reductions following the failure of its Angelman syndrome treatment.
BIOBOOK TEAM
biobookmedia@biobook.co.krRoche’s New Drug for Uveitic Macular Edema Faces FDA Decision in July 2027
BMS Reports Concurrent Phase 3 Success in Idiopathic Pulmonary Fibrosis and Solid Tumors
Merck’s $1.3 trillion eye disease drug raises safety and efficacy concerns: “Limited competitiveness”
CHA Biotech Holds 24.23% Stake in CMG Pharmaceutical as Share Consolidation Reduces Its Share Count
Rural Development Administration Develops Two Molecular Markers to Identify Leaf Splitting in Squash
MFDS Begins Distribution of Internationally Certified Reference Materials, Including 24 New Items, for a Total of 882
