Novartis’s Narasimhan faces his first big test
Revelations of data manipulation might not be fatal, but they will hurt the chief exec’s attempted rebranding of Novartis as a more ethical pharma company.
Threats to Spinraza are mounting, but Biogen still has cards to play
Competition is looming from Novartis and Roche but the latest results from Spinraza show that Biogen still has a strong case to make for the SMA therapy.
PPMD 2019 – Pfizer’s Duchenne gene therapy halt is good news for Sarepta
Worries over toxicity with Pfizer's PF-06939926 halts trial dosing and could leave the way clear for its DMD rival Sarepta.
Krystal plays down dropout to claim a mid-stage win
Despite concerns about a missing patient, results from a tiny trial in epidermolysis bullosa boosted Krystal Biotech’s stock by 41%.
Vertex’s second transformation: Duchenne time
The cystic fibrosis player does deals with Crispr and Exonics to launch itself into a highly competitive rare disease area.
Upcoming events – Protagonist and Pfizer target rare diseases
Protagonist Therapeutics and Pfizer will soon get an idea of their respective chances in beta-thalassaemia and Duchenne muscular dystrophy.
Biomarin suffers minimal bleeding on valrox readout
Three-year data from Biomarin’s haemophilia A gene therapy fail to quash concerns about durability, but the company insists that the results are good enough to file on.
Zolgensma heads up spate of US approvals
The US FDA granted seven approvals in two days last week, including an eagerly awaited thumbs-up for Novartis’s gene therapy Zolgensma.
Biomarin hopes long-term valrox data can stem the bleeding
Expectations are low for a three-year update on the group’s haemophilia A gene therapy, and Biomarin’s rivals could capitalise on any slip-up.
Kickstarting ideas
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