Ultragenyx Pharmaceutical Inc.

ultragenyx.com

What Do They Do?

Develops and sells medicines for serious rare and ultra-rare genetic diseases, serving patients with those conditions.

How Do They Make Money?

Sells approved medicines through distributors, with sales counted when products are delivered; it also earns Crysvita royalties as the related sales occur.

FY2025 revenue by product or service revenue

Segment	Share	How money arrives
Collaboration and license	0.0% FY2022: 61%
Product sales Sold outright	55% FY2022: 33%	Sold outright
Royalty Licensed	45% FY2022: 6.0%	Licensed

Who Are Their Customers?

The company sells approved medicines through a limited number of distributors; its customers also include collaboration partners, drug wholesalers and retail pharmacy distributors.

Customers above 10% of revenue, FY2025

of revenue from one customer

Do Revenues Turn Into Profits?

No. It lost $575M in FY2025.

Operating income, FY2025

−79% of revenue

▼ 0% (Loss) vs FY2024 (−$536M, −96%)

Net income, FY2025

−85% of revenue

▲ +1% (Loss) vs FY2024 (−$569M, −102%)

Free cash flow, FY2025

−70% of revenue

▲ +12% (Loss) vs FY2024 (−$421M, −75%)

How Are They Changing?

Since the FY2025 annual report

Latest quarter: Q2 FY2026, the three months to Jun 30, 2026

Revenue: ▲ +28%; vs $167M in Q2 FY2025
Net income: ▼ −20% (Loss); vs −$115M in Q2 FY2025

Product Revenue share of revenue vs 48.5% in Q2 FY2025

Sep 2, 2026 Phase 3 Aspire study missed its main efficacy measures The company said it will review the apazunersen program and assess significant expense reductions.
Aug 19, 2026 FDA grants accelerated approval for GENGLYCOS
Aug 11, 2026 Settled litigation with a generic Dojolvi applicant The license allows generic marketing beginning January 1, 2033, subject to conditions; litigation with other applicants remains pending.
Aug 4, 2026 Reaffirmed 2026 revenue and expense guidance Reaffirmed revenue guidance of $730 million to $760 million and forecast combined research and selling, general and administrative expenses flat to slightly down versus 2025.

Growing. Revenue grew 92% in 4 years, from $351.4M in FY2021 to $673M in FY2025, about 18% a year.

Crysvita revenue model The older report presents Crysvita as a global collaboration; the latest says the shared-sales period ended and Crysvita revenue shifted to royalties tied to underlying sales.

Evkeeza markets The older report describes Evkeeza as approved in the U.S. and EEA; the latest also lists Brazil, Mexico and Japan among approved markets.

Revenue over time

Collaboration and license Product sales Royalty

$1B$500M$0

FY2021 FY2022 FY2023 FY2024 FY2025

Who Do They Compete With?

Competes with Arrowhead Pharmaceuticals, Eli Lilly, Dicerna, Novo Nordisk, CRISPR Therapeutics, Ionis, Oak Hill Bio, Neuren Pharmaceuticals, MavriX Bio, Esteve, Orchard Therapeutics, Denali, JCR Pharma, Beam Therapeutics, Moderna, Arcturus Therapeutics, Bloomsbury, iECURE, Camp 4, and Monopar Therapeutics

Comments