Bristol Myers Squibb will boost its cell therapy manufacturing network with in-house viral vector production thanks to the acquisition of Novartis’ Libertyville, Illinois facility.
Novartis has presented new data which ‘underscore the transformational and sustained benefit’ of spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec): with treated children maintaining motor milestones after 7.5 years.
Zolgensma has received a recommendation for approval despite running into problems recently, including manufacturing questions and data integrity issues.
US FDA places a hold on a clinical study testing Novartis’ Zolgensma in patients with SMA Type 2, after information provided to the authorities by AveXis.
Novartis announces its third quarter results that show Zolgensma is outperforming sales expectations, but company notes approval in EU and Japan pushed back over manufacturing queries.
Alleged manipulated and potentially inaccurate clinical data in the BLA for the $2.1m Zolgensma spark possibility for criminal penalties against AveXis.
Bluebird has received EU conditional marketing approval for patients with transfusion-dependent β-thalassemia, becoming the first treatment from its pipeline to receive approval.
Novartis receives US FDA approval for its gene therapy, Zolgensma, which is a treatment for spinal muscular atrophy, and prices the drug at $2.1m per patient.
