BioMarin Pharmaceutical is to buy rare disease-focused Prosensa for $680m in cash and $160m in contingent milestones if Prosensa's Duchenne muscular dystrophy product – the GSK-reject drisapersen – is approved quickly in the US and Europe.
BioMarin Pharmaceutical is to buy rare disease-focused Prosensa for $680m in cash and $160m in contingent milestones if Prosensa's Duchenne muscular dystrophy product – the GSK-reject drisapersen – is approved quickly in the US and Europe.
Drisapersen had been partnered with GlaxoSmithKline but the big pharma pulled out of the deal following drisapersen's failure in the...
Just days after Roche stopped shipments of the DMD gene therapy following safety concerns raised by two patient deaths, its EU approval application has hit a block.
Seven weeks before its action date, Capricor got an FDA complete response for cell therapy deramiocel in DMD-related cardiomyopathy. It has a Phase III study nearing readout, though.
The osteogenesis imperfecta drug stumbled in a second interim analysis.
The oral, twice-daily candidate was expected to control decline in motor function in DMD patients regardless of their gene mutations.
The BRUIN CLL-314 trial was designed to show noninferiority, but the p-value indicated a favorable result for Jaypirca in both the pre-treated and intent-to-treat populations.
Its multibillion dollar investment demonstrates the importance of the US to AstraZeneca, with half of its targeted $80bn revenues by 2030 set to come from the country, Pascal Soriot said as it presented strong second-quarter results.
The company is getting just $55m from the sale of Tecelra and a couple of late-stage T-cell therapies to US WorldMeds.
