Key Takeaways
Scrip interviewed Ultragenyx founder and CEO Emil Kakkis about an upcoming Phase III data read out, the upcoming launch of its first gene therapy and business strategy.
The company is on a notable growth trajectory and aiming to reach profitability in 2027.
Investors are eagerly awaiting an interim analysis from a Phase III trial for setrusumab in osteogenesis imperfecta, which could be a big future growth driver.
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
Ultragenyx is on a notable growth trajectory fueled by commercial drugs Crysvita (burosumab) for X-linked hypophosphatemia (XLH) and Dojolvi (triheptanoin) for long-chain fatty acid oxidation disorders (LC-FAOD), and new launches are expected to continue