Is RARE a Buy? What to Consider in 2026
Short answer
The bull case for Ultragenyx Pharmaceutical (RARE) rests on Near-term gene therapy approvals: Two gene therapies are under FDA review with 2026 decision dates: DTX401 for glycogen storage disease type Ia and UX111 for Sanfilippo syndrome type A. Revenue (TTM) is ~$670M. If you believe that thesis holds, the real questions become position sizing and overlap, not timing. The main risk to that view: Ultragenyx remains unprofitable, with a net loss of about $575 million in 2025 and roughly $737 million of cash as of December 2025, so continued spending or delayed approvals could pressure the balance sheet and raise dilution or financing risk. Whether RARE is a buy comes down to whether you believe the thesis. This is informational, not a recommendation, and Walnut is not an investment adviser.
Ultragenyx Pharmaceutical (NASDAQ: RARE) develops and sells treatments for rare and ultra-rare genetic diseases, conditions with small patient populations and often no approved therapy. Its commercial base includes Crysvita for X-linked hypophosphatemia (its largest product), Dojolvi for long-chain fatty acid oxidation disorders, Evkeeza, and Mepsevii, which together produced about $673 million of revenue in 2025. The company spans multiple modalities, protein-replacement, small molecule, antisense, and gene therapy, and is preparing several late-stage programs for potential approval. The investment picture is that of a growing but still loss-making specialty biotech. Revenue grew roughly 20% in 2025, yet the company posted a net loss of about $575 million as it funds heavy R&D. Management has reaffirmed 2026 revenue guidance of $730 million to $760 million and reiterated a goal of reaching profitability in 2027, contingent on commercial growth, expense discipline, and monetizing potential priority review vouchers. The stock therefore trades largely on pipeline catalysts and the credibility of that profitability timeline rather than on current earnings.
What's the case for buying RARE?
1. Near-term gene therapy approvals
Two gene therapies are under FDA review with 2026 decision dates: DTX401 for glycogen storage disease type Ia and UX111 for Sanfilippo syndrome type A. Both target diseases with no approved therapies, giving Ultragenyx a potential first-mover position. Approvals could also unlock sellable priority review vouchers that management is counting on for cash.
2. Angelman syndrome readout
Pivotal Phase 3 Aspire data for GTX-102 in Angelman syndrome is expected in the second half of 2026. Angelman is a large opportunity relative to Ultragenyx's typical ultra-rare markets, so a positive readout would materially expand the addressable pipeline. It is also one of the highest-risk binary events on the calendar.
3. Growing commercial base
Crysvita, guided to roughly $500 million to $520 million in 2026, anchors a portfolio that management expects to keep growing at a high-single to low-double-digit rate. A steady base business helps fund the pipeline and supports the stated 2027 profitability target. Dojolvi and Evkeeza add incremental, if smaller, contribution.
4. Cost discipline toward profitability
Ultragenyx cut about 10% of its workforce and guided 2026 R&D and SG&A to be flat-to-down as it concentrates resources on near-term launches. The restructuring is meant to preserve cash and make the 2027 profitability goal credible. Execution on this cost curve is central to the equity story.
What are the risks to RARE?
Ultragenyx remains unprofitable, with a net loss of about $575 million in 2025 and roughly $737 million of cash as of December 2025, so continued spending or delayed approvals could pressure the balance sheet and raise dilution or financing risk. The value concentrated in binary regulatory and clinical events (DTX401, UX111, and especially the GTX-102 Angelman readout) means a single negative outcome could sharply move the stock. Rare-disease revenue depends on small patient counts, payer reimbursement, and diagnosis rates, all of which can disappoint. The company faces well-capitalized competitors in gene therapy and orphan drugs, including BioMarin, Sarepta, and larger players. Finally, the 2027 profitability target is a management objective, not a guarantee, and could slip.
How is RARE valued? (as of MAY 2026)
Snapshot for RARE as of July 2026, sourced from Yahoo Finance and may be delayed. Valuation figures move with price and earnings; verify the current numbers with your broker before deciding.
- Revenue (TTM): ~$670M
- FY2025 revenue: ~$673M (up ~20%)
- Q1 2026 revenue: ~$136M
- FY2025 net loss: ~$575M
- Cash and marketable securities: ~$737M (Dec 2025)
- Market cap: ~$2.6B
Ultragenyx trades at a modest multiple of revenue for a biopharma but has no earnings, so conventional P/E valuation does not apply. The market is effectively pricing the pipeline and the credibility of the 2027 profitability goal. Guidance for 2026 revenue is $730 million to $760 million, which excludes any potential new product launches.
How do you decide if RARE is a buy?
Rather than asking whether RARE is a buy in the abstract, it tends to help to answer four questions:
- Thesis: do you believe the case above, and is it still true today?
- Time horizon: a single stock can be volatile, so a longer horizon absorbs more of the swings.
- Position sizing: a thesis can be right and the sizing still wrong; decide how much of your portfolio one name should be.
- Overlap: check whether you already hold RARE indirectly through an index or sector ETF before adding more.
For the full picture, see the RARE stock guide (what the company does, the ETFs that hold it, similar stocks, and the themes it fits). In Walnut you can ask its AI about RARE against your real portfolio and see your actual exposure before deciding.
The bottom line on RARE
The bottom line: Ultragenyx Pharmaceutical's story right now is Near-term gene therapy approvals, with revenue (ttm) at ~$670M. If you believe that narrative continues, the call is about sizing RARE sensibly and checking overlap with what you own; if you doubt it (the risk: ultragenyx remains unprofitable, with a net loss of about $575 million in 2025 and roughly $737 million of cash as of December 2025, so continued spending or delayed approvals could pressure the balance sheet and raise dilution or financing risk.), it is not for you. Decide from the thesis, not the ticker. Walnut is not an investment adviser.
Build a basket around RARE with Walnut
Use Ultragenyx Pharmaceutical as one constituent in a thematic basket Walnut's AI helps you assemble. Describe a thesis you believe in, the AI proposes the holdings and weights, and you approve before any broker order.
FAQ
Is RARE a good stock to buy right now?
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The case for Ultragenyx Pharmaceutical right now is Near-term gene therapy approvals, with revenue (ttm) at ~$670M. If you believe that thesis holds, RARE is a way to own it and the real questions are sizing and overlap, not timing; the main risk to that view is ultragenyx remains unprofitable, with a net loss of about $575 million in 2025 and roughly $737 million of cash as of December 2025, so continued spending or delayed approvals could pressure the balance sheet and raise dilution or financing risk. So it comes down to whether you believe the thesis. Walnut is not an investment adviser and this is not a recommendation.
What does Ultragenyx Pharmaceutical do?
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Ultragenyx Pharmaceutical (NASDAQ: RARE) develops and sells treatments for rare and ultra-rare genetic diseases, conditions with small patient populations and often no approved the
What are the main risks of RARE?
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Ultragenyx remains unprofitable, with a net loss of about $575 million in 2025 and roughly $737 million of cash as of December 2025, so continued spending or delayed approvals could pressure the balance sheet and raise dilution or financing risk. The value concentrated in binary regulatory and clinical events (DTX401, UX111, and especially the GTX-102 Angelman readout) means a single negative outcome could sharply move the stock. Rare-disease revenue depends on small patient counts, payer reimbursement, and diagnosis rates, all of which can disappoint. The company faces well-capitalized competitors in gene therapy and orphan drugs, including BioMarin, Sarepta, and larger players. Finally, the 2027 profitability target is a management objective, not a guarantee, and could slip.
What does Ultragenyx do?
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Ultragenyx develops and commercializes treatments for rare and ultra-rare genetic diseases. It sells four approved products, led by Crysvita, and is advancing several late-stage gene therapies and other candidates across metabolic, neurologic, and other disorders.
Is Ultragenyx profitable?
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No. As of May 2026 Ultragenyx was not profitable, reporting a net loss of about $575 million in 2025 as it funds heavy research and development. Management has stated a goal of reaching profitability in 2027, though that is an objective rather than a certainty.
How much revenue does Ultragenyx generate?
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Full-year 2025 revenue was about $673 million, roughly 20% growth, and first-quarter 2026 revenue was about $136 million. The company reaffirmed 2026 guidance of $730 million to $760 million, which excludes any potential new product launches.
What is Ultragenyx's biggest product?
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Crysvita, a treatment for X-linked hypophosphatemia, is its largest product, with 2026 revenue guided to roughly $500 million to $520 million. Dojolvi, Evkeeza, and Mepsevii make up the rest of the commercial portfolio.
Walnut is informational and is not an investment adviser. This page is educational and not a recommendation to buy or sell RARE; figures are approximate and dated, and your own situation, time horizon, and risk tolerance should drive any decision. Verify current data before investing.