BioMarin has yet to treat a patient with Roctavian, the first ever hemophilia A gene therapy, eight months after its first approval in Europe, as pricing delays cloud its commercial prospects.
BioMarin has hit a serious delay to agreeing a price for its hemophilia A gene therapy, Roctavian, in Germany, the product’s first global launch market.
The company gained a conditional marketing approval from the European Medicines Agency for Roctavian (valoctocogene roxaparvovec) on 24 August last year,
Plus deals involving J&J/Addex/Sinntaxis, Concentra/Kronos, Granata/Oviva, Coeptis/Z Squared, Ligand/Channel and more.
The company plans to expand manufacturing of the HER2-targeting ADC beyond China and plans to file for US FDA approval in 2025.
PTC said its updated Phase II data for PTC 518 met the primary endpoint of Huntingtin’s protein reduction, but analysts said there isn’t clear correlation with clinical benefit.
Restructuring Edition: Unity unveiled more Phase IIb data for lead asset UBX1325 along with plans to shed its workforce while seeking a partner for it and other drugs. Also, Ono is consolidating US employees at one site, Entrada is cutting its workforce by 20% and other recent strategic shifts.
Restructuring Edition: Unity unveiled more Phase IIb data for lead asset UBX1325 along with plans to shed its workforce while seeking a partner for it and other drugs. Also, Ono is consolidating US employees at one site, Entrada is cutting its workforce by 20% and other recent strategic shifts.
Genmab will submit an sBLA for Epkinly in R/R follicular lymphoma in H1 2025, backed by positive Phase III data.
Drugmakers aren’t expecting a big financial hit from tariffs for now, but a report commissioned by PhRMA suggests the cost of pharma-sector tariffs could be steep.
