Marin County’s Ultragenyx Pharmaceutical estimates sales grew nearly 20% in 2023

Ultragenyx Pharmaceutical Inc. (Nasdaq: RARE), a Marin County-based developer and marketer of treatments for serious rare and ultrarare genetic diseases, this week said that it wrapped 2023 likely with a double-digit annual jump in revenue as sales continue to rise for its two main products.

The Novato company on Sunday released preliminary 2023 revenue and cash-use estimates as well as 2024 projections. Last year, the company took in $430 million–$435 million. That would be an 18.4%–19.7% increase from $363.3 million in reported for 2022, which itself was 3% higher than in 2021.

Revenue last year from Crysvita, a treatment for treatment of XLH, a hereditary, progressive and lifelong musculoskeletal disorder affecting 48,000 worldwide, was estimated to be $325 million–$330 million. That would be up 26.1%–28% from reported 2022 revenue of $257.7 million ($215 million in licensing fees plus $42.7 million in product sales).

Dojolvi is a treatment for rare metabolic diseases that prevent the conversion of fat into energy, potentially causing low blood sugar, muscle rupture, and heart and liver disease in 8,000–14,000 people globally. Sales last year were figured to be $70 million–$71 million, which would be up 25.9%–27.7% from $55.6 million reported in 2022.

The company expects to report full annual and quarterly results next month.

Revenue this year is expected to total $500 million–$530 million, with Crysvita sales of $375 million–$400 million and Dojolvi of $75 million–$80 million.

“2024 is poised to be a year of significant momentum for Ultragenyx, with clinical catalysts across multiple value-driving programs, meaningful revenue growth from our commercial products, and continued focus on financial discipline,” said Emil D. Kakkis, M.D., Ph.D., founder, CEO and president, in a news release. “We anticipate several important milestones in the first half of the year, including completing enrollment of our Phase 3 studies in osteogenesis imperfecta, interim data on a substantive portion of patients enrolled in our Phase 1/2 study in Angelman syndrome, Stage 1 data from our pivotal Phase 1/2/3 study in Wilson disease, and topline Phase 3 data from our GSDIa gene therapy program.”

In June, Ultragenyx celebrated the opening its new gene therapy production facility in Bedford, Massachusetts. The company launched into gene therapy with the purchase of Dimension Therapeutics in 2015, and the Boston area became the firm’s base for development of these treatments.

The 110,000-square-foot plant employs 120 and is set to significantly scale up what’s possible in manufacturing such treatments, the company said. Ultragenyx also has facilities on the San Francisco peninsula and elsewhere in the Boston area.

Here’s a video overview of the new Bedford facility:

Kakkis founded Ultragenyx in April 2010. The company has several treatments in varying phases of development and testing.

Jeff Quackenbush covers wine, construction and real estate. Reach him at jquackenbush@busjrnl.com or 707-521-4256.