Drug Approval | February 24, 2026
Ultragenyx bags FDA priority review for groundbreaking GSDIa gene therapy
If approved, DTX401 would be the first treatment to address the disease at its root cause
If approved, DTX401 would be the first treatment to address the disease at its root cause
UBT251 appeared to have a safe and well-tolerated profile consistent with incretin-based therapies
The clinical program has benefited from extensive European Union support, including the IMI2 TRIC-TB and UNITE4TB projects, as well as funding from the EDCTP2 bEto-TB project
The alliance directly links VivaMed’s computational drug hypotheses with Syngene’s preclinical development capabilities
The project entails a total planned capital expenditure of approximately Rs. 500 - 520 crore
BLA is based on a robust data package supporting similarity to Orencia (abatacept) IV for infusion through totality of evidence including pharmacokinetic (PK) data
Both non-transfusion-dependent and transfusion dependent cohorts met their respective primary and secondary endpoints
Our vision is to position India not just as a consumer of advanced simulation technologies but as a creator and exporter of them
The drug’s safety profile in LIBRETTO-432 aligned with previous selpercatinib studies, showing no unexpected concerns
Expansion at the Oss (NL) site adds capacity and R&D capabilities, supporting the development and manufacturing of ADCs and other novel bioconjugates from First-in-Human to commercialization
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