Vertex CEO Jeffrey Leiden has offered an olive branch to negotiators in the UK that could see the cystic fibrosis drug Orkambi finally supplied to patients in England.
European regulators have recommended a licence for Akcea and Ionis’ ultra-rare disease drug Waylivra, a therapy that the FDA rejected for the US market last year.
In this issue of Deep Dive we look at innovation in pharma R&D, asking how can pharmaceutical companies change up the development model with new strategies and technologies to cut waste
To have any impact, patient engagement needs to start with the drug development pathway, not at the point of licensing. That’s especially true in the rare disease space, where the unme
The FDA has granted a fast review for Alexion Pharmaceuticals’ filing for its Soliris, as a treatment for a rare neurological disease that leads to worsening disability, blindness, paralysi