NOVATO, Calif. - Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, reported preliminary unaudited 2021 Crysvita revenue in Ultragenyx territories and Dojolvi global revenue, cash and investments at year end 2021, and provided 2022 revenue guidance for Crysvita in Ultragenyx territories and Dojolvi globally.

'We are seeing strong growth for Crysvita multiple years out from launch and expect similar progress for Dojolvi heading into year two,' said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. '2022 will be a year of execution and momentum for us, both on the commercial side as we gear up for the launch of Evkeeza in Europe and other key ex-US geographies, and on the clinical side as we progress our large and late-stage pipeline that includes four pivotal programs.'

Ultragenyx will present at the 40?? annual J.P. Morgan Healthcare Conference on Monday, January 10, 2022 at 3:45 p.m. ET. The live and archived webcast of the presentation will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm.

Financial Update

Crysvita: 2021 Preliminary Revenue (unaudited) and 2022 Guidance

Crysvita revenue in Ultragenyx territories* for the year ended December 31, 2021 is approximately $191 million to $193 million. This is above the guidance range of $180 million to $190 million that was provided at the beginning of 2021, notwithstanding disruptions and effects from the COVID-19 pandemic. For 2022, Crysvita revenue in the Ultragenyx territories is estimated to be between $250 million and $260 million, representing growth of 33% year-over-year at the mid-point of our guidance.

Dojolvi: 2021 Preliminary Revenue (unaudited) and 2022 Guidance

Dojolvi revenue for the year ended December 31, 2021 is approximately $38 million to $40 million. For 2022, Dojolvi revenue is estimated to be between $55 million and $65 million, representing growth of 60% year-over-year at the mid-point of our guidance in the second year of launch.

2021 Ending Cash Position (unaudited)

Cash, cash equivalents, and available-for-sale investments were approximately $1.0 billion as of December 31, 2021.

The 2021 revenue and cash position included in this release are preliminary and prior to the completion of review and audit procedures by Ultragenyx's external auditors and are therefore subject to adjustment. The preliminary revenue results are based on management's initial analysis of operations for the quarter and year ended December 31, 2021. The Company expects to issue full financial results for the fourth quarter and fiscal year 2021 in February 2022.

Recent Updates and 2022 Milestones

Evkeeza for Homozygous Familial Hypercholesterolemia (HoFH): Submission of reimbursement dossiers expected in European countries in 2022

Ultragenyx and Regeneron announced a license and collaboration agreement for Ultragenyx to commercialize and distribute Evkeeza (evinacumab) in countries outside of the U.S. This includes the European Economic Area where Evkeeza was approved in June 2021 as a first-in-class monoclonal antibody targeting ANGPTL3 for use together with diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies to treat adults and adolescents aged 12 years and older with HoFH.

Ultragenyx plans to submit reimbursement dossiers with national health authorities in Europe in 2022.

GTX-102 for Angelman Syndrome: Phase 1/2 study is titrating patients in Canada and the U.K. with data anticipated mid-year 2022

The first four patients in the Phase 1/2 study have received multiple doses of GTX-102 and regular assessments for safety. To date three have also received a preliminary assessment of clinical response. There have been no treatment-related serious adverse events of any type nor adverse events related to lower extremity weakness observed in these patients, and initial assessments have shown early signs of clinical activity.

The data safety monitoring board (DSMB) met to discuss the assessments for the first two patients in Cohort 4 (ages 4 to

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