Ultragenyx: Commercial Engine Fuels Rare Disease Pipeline

  • Ultragenyx shows strong revenue growth and disciplined expense management, making it a compelling long-term investment despite inherent biotechnology risks. The company's diverse revenue streams from Crysvita, Dojolvi, and Evkeeza fund innovation and buffer against financial volatility. Key catalysts include the FDA's Priority Review of UX111 for Sanfilippo syndrome type A, with a PDUFA date set for August 2025.