The ultra-rare disease specialist Ultragenyx is approaching several important catalysts this year, including US Food and Drug Administration action on UX111 – the firm’s first gene therapy, which targets Sanfilippo syndrome – and a pivotal Phase III data readout on setrusumab for osteogenesis imperfecta (OI), a drug that could become the company’s biggest revenue generator yet.
Ultragenyx CEO Emil Kakkis: ‘Our Future Is In Our Hands’
The founder and CEO of Ultragenyx talked with Scrip as the company approaches a pivotal data readout and awaits US FDA action on its first gene therapy.
