Ultragenyx Pharmaceutical Inc. (RARE): BCG Matrix [Dec-2025 Updated]

You're looking for a clear, no-nonsense breakdown of Ultragenyx Pharmaceutical Inc.'s (RARE) portfolio as of late 2025, and the BCG Matrix is the perfect tool to map their commercial products against their high-risk, high-reward pipeline. Honestly, the picture shows a company leaning heavily on Crysvita as the clear Star, supported by Dojolvi as a reliable Cash Cow, while the small Mepsevii product barely registers as a Dog. The crucial tension, though, is that these products are funding the massive R&D spend causing the $180 million Q3 net loss, all aimed at turning pipeline Question Marks like UX143 and GTX-102 into future Stars that can push total revenue past the projected $640 million to $670 million range. Let's dive into where you should be focusing your attention.

Background of Ultragenyx Pharmaceutical Inc. (RARE)

You're looking at a company that started with a very specific, high-stakes focus. Ultragenyx Pharmaceutical Inc. was founded in 2010 in Novato, California, by Emil D. Kakkis, M.D., Ph.D. The core mission driving Ultragenyx Pharmaceutical Inc. is to develop and commercialize novel products for the treatment of serious rare and ultra-rare genetic diseases, targeting conditions that often lack any approved therapies. This focus places the company squarely in the high-risk, high-reward orphan drug space.

Ultragenyx Pharmaceutical Inc. employs a strategy that utilizes multiple drug modalities, including biologics, small molecules, gene therapies, ASO, and mRNAs. The company's current commercial success, which funds its extensive research and development, is anchored by key approved products. For instance, Crysvita (burosumab-twza), used for X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia (TIO), is a cornerstone therapy. The company also commercializes Dojolvi (triheptanoin).

As of late 2025, Ultragenyx Pharmaceutical Inc. is projecting continued revenue expansion from these commercial products. The reaffirmed total revenue guidance for the full year 2025 sits between $640 million and $670 million, representing an expected growth of 14-20% compared to 2024. Specifically, Crysvita revenue for 2025 is guided to be between $460 million and $480 million, while Dojolvi is guided for $90 million to $100 million. Still, the company is not yet profitable, reporting a net loss of $180 million for the third quarter of 2025 alone.

To support its late-stage pipeline, Ultragenyx Pharmaceutical Inc. took a strategic financial step in November 2025, bolstering its balance sheet with $400 million from the sale of a portion of its Crysvita royalty interest. This capital is intended to support pivotal milestones, including expected Phase 3 data readouts for UX143 in osteogenesis imperfecta around the end of 2025, and progress on gene therapies like UX111 for Sanfilippo syndrome, which had a PDUFA action date of August 18, 2025. The company is working toward a goal of achieving full-year GAAP profitability by 2027.

Ultragenyx Pharmaceutical Inc. (RARE) - BCG Matrix: Stars

The business unit considered a Star for Ultragenyx Pharmaceutical Inc. is Crysvita (burosumab). The full year 2025 revenue guidance for Crysvita is reaffirmed to be in the range of $460 million to $480 million. This product contributes significantly to the total projected 2025 revenue for Ultragenyx Pharmaceutical Inc., which is set between $640 million to $670 million, representing an approximate growth of 14-20% compared to 2024. The performance across the first three quarters of 2025 demonstrates this high-growth trajectory.

The product maintains a dominant position in the market for X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia (TIO). North America accounts for around 40% of the Crysvita Injection market, with approximately 25% of patients in North America diagnosed with XLH being treated with Crysvita Injection. The continued investment in global penetration is supported by strong regional performance.

• Crysvita is approved in the U.S. for the treatment of X-linked hypophosphatemia (XLH) and FGF23-related hypophosphatemia in tumor induced osteomalacia (TIO).
• Latin America and Türkiye product sales in Q1 2025 grew 52% compared to Q1 2024.
• Total Crysvita revenue for the nine months ended September 30, 2025, reached $335,214 thousand.
• The ultra-orphan disease market segment requires ongoing support for global penetration.

Ultragenyx Pharmaceutical Inc. (RARE) - BCG Matrix: Cash Cows

Cash Cows are business units or products with a high market share but low growth prospects. Ultragenyx Pharmaceutical Inc. positions certain commercialized therapies here, as they generate more cash than they consume, which is critical for funding the company's more speculative Question Marks and Stars.

Dojolvi (triheptanoin) fits this profile well, being an established, first-in-class therapy for long-chain fatty acid oxidation disorders (LC-FAOD). This product provides a stable, predictable revenue stream that helps fund the company's extensive R&D pipeline. For the full year 2025, Ultragenyx Pharmaceutical Inc. expects Dojolvi revenue to be in the range of $90 million to $100 million. This expected revenue range represents a lower growth rate, projected at 2-14% year over year for 2025.

To illustrate the current performance supporting this classification, look at the recent quarterly results. Dojolvi product sales for the third quarter of 2025 were $24.3 million, marking a year-over-year increase of 14%. Because this market is mature for this specific indication, the investment in promotion and placement is relatively contained, allowing the product to 'milk' its gains passively.

The characteristics of these Cash Cows can be summarized against the backdrop of the company's overall 2025 financial outlook. Investments into supporting infrastructure for these established products are focused on efficiency gains to further increase cash flow, which is exactly what you want from a Cash Cow.

Evkeeza (evinacumab), while technically a smaller contributor compared to the primary revenue driver, Crysvita, acts as a secondary cash generator, especially as Ultragenyx Pharmaceutical Inc. expands its commercial footprint outside the U.S. The growth trajectory for this ex-U.S. business is notable; Evkeeza recorded sales of $16.7 million in the third quarter of 2025, which was an increase of 57% compared to the prior year period, driven by launches in Ultragenyx Pharmaceutical Inc.'s territories outside the United States.

These products are the foundation that supports the entire enterprise. You need this reliable cash flow to cover administrative costs and fund the riskier ventures in the pipeline. Here's the quick math: the expected $90 million to $100 million from Dojolvi, combined with the growing international sales of Evkeeza, provides the necessary ballast.

The role of these Cash Cows in the Ultragenyx Pharmaceutical Inc. portfolio is clear:

• Provides a stable, predictable revenue stream.
• Helps fund the company's extensive R&D pipeline.
• Generates cash to cover corporate administrative costs.
• Supports the path to full year GAAP profitability in 2027.

What this estimate hides is the potential for Dojolvi's growth rate to hit the high end of the 14% range, which would make it a stronger performer than currently implied by the low-growth Cash Cow designation, but still not a Star given the market maturity. Finance: draft 13-week cash view by Friday.

Ultragenyx Pharmaceutical Inc. (RARE) - BCG Matrix: Dogs

You're looking at the products that aren't driving significant growth or market leadership for Ultragenyx Pharmaceutical Inc. (RARE) right now. These are the Dogs in the Boston Consulting Group (BCG) Matrix framework.

For Ultragenyx Pharmaceutical Inc. (RARE), Mepsevii (vestronidase alfa-vjbk) fits this profile. It's the smallest commercial product in the portfolio, specifically targeting the ultra-rare condition Mucopolysaccharidosis VII (MPS VII). Its performance metrics suggest it resides firmly in the low growth, low market share quadrant.

Here's a look at the financials for this unit as of the third quarter of 2025. The revenue came in at $7 million for Q3 2025. That figure represents a year-over-year decline of 27%, showing negative momentum in a segment that isn't expanding rapidly.

The contribution of Mepsevii is marginal when you compare it to the company's overall expected performance. Ultragenyx Pharmaceutical Inc. (RARE) projects total revenue for the full year 2025 to be between $640 million and $670 million. Honestly, the cash tied up here is likely minimal, but the return is almost negligible too.

The typical strategy for a Dog is to minimize exposure, as expensive turn-around plans rarely pay off in these situations. You want to avoid sinking more capital into a product with low market share in a slow-growth area.

The characteristics defining Mepsevii as a Dog include:

• Low market share in its specific indication.
• Operating in a slow-growth market segment.
• Requiring minimal, yet consistent, maintenance investment.
• Marginal impact on total projected 2025 revenue.

To put the revenue contribution into perspective, here is a simple comparison:

This product is definitely a candidate for divestiture or harvesting its remaining value, depending on the strategic view of maintaining a presence in the MPS VII space. Its current trajectory suggests it neither earns nor consumes significant cash, but it certainly isn't a source of funding for the Stars or Cash Cows.

Finance: draft divestiture analysis for Mepsevii by end of Q4 2025.

Ultragenyx Pharmaceutical Inc. (RARE) - BCG Matrix: Question Marks

You're looking at the pipeline assets that are currently consuming significant cash while holding the potential to become the next generation of revenue drivers for Ultragenyx Pharmaceutical Inc. These are the classic Question Marks: high-growth markets, but the company hasn't secured a dominant market share yet-in fact, for most, market share is currently zero because they await approval. This phase demands heavy investment, which is clearly reflected in the recent financials.

The financial reality of supporting these high-potential programs is evident in the third quarter of 2025. Ultragenyx Pharmaceutical Inc. reported a net loss of $180 million for the three months ended September 30, 2025. The total operating expenses for that quarter hit $331 million, which included $216 million dedicated to Research and Development (R&D). This R&D spend, along with $87 million in investments for pre-launch inventory manufacturing, is the cash burn fueling these Question Marks. Net cash used in operations for Q3 2025 was $91 million, illustrating the current negative return on these assets.

Here's a look at the specific programs driving this investment profile:

• UX143 (setrusumab) for Osteogenesis Imperfecta (OI): Pivotal Phase 3 data from the Orbit and Cosmic studies are expected around the end of 2025. This represents a massive market opportunity in a rare genetic bone disorder affecting approximately 60,000 people globally with no approved treatments. The Orbit study has a pre-specified $\text{p}$ value threshold of $\text{p}<\text{0.04}$ for its primary endpoint of annualized fracture rate.
• GTX-102 for Angelman Syndrome: Phase 3 Aspire study enrollment was completed in July 2025, involving approximately 129 participants aged 4 to 17 with a full maternal UBE3A gene deletion. Data from this study are anticipated in the second half of 2026.
• UX111 (gene therapy) for Sanfilippo Syndrome Type A: This is a high-risk, high-reward gene therapy that received a Complete Response Letter (CRL) from the FDA due to Chemistry, Manufacturing, and Controls (CMC) observations. Ultragenyx Pharmaceutical Inc. plans to resubmit the Biologics License Application (BLA) in early 2026, with potential approval now delayed to 2026. The FDA acknowledged the robustness of the clinical and biomarker data.
• DTX401 (gene therapy) for Glycogen Storage Disease Type Ia (GSDIa): The rolling BLA submission was initiated in August 2025, with completion expected in the fourth quarter of 2025. The BLA includes 96-week Phase 3 data showing patients in the ongoing DTX401 group had a 60% reduction from baseline in total daily cornstarch, and the crossover group showed a 64% reduction. This therapy targets a condition affecting about 6,000 people in commercially accessible regions.

These programs are the primary drivers of the Q3 2025 net loss of $180 million, requiring heavy R&D spend for future growth. The strategy here is clear: invest heavily now to quickly gain market share and convert these Question Marks into Stars, or face them becoming Dogs if the market opportunity is missed or the investment fails to yield approval.

Here is a summary of the key pipeline milestones and associated financial context:

The company's cash position as of September 30, 2025, was $447 million in cash, cash equivalents, and securities, which was bolstered by a $400 million Crysvita royalty transaction announced the same day. This financing is defintely intended to bridge the gap until these Question Marks mature into revenue-generating Stars.