Investor-Grade Analysis | March 2026
Ada Cockpit Research
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) is a biopharmaceutical company focused on developing and commercializing therapies for rare and ultra-rare genetic diseases. Founded in 2010 by Emil D. Kakkis, M.D., Ph.D., the company is headquartered in Novato, California.
| Metric | Value | Notes |
|---|---|---|
| Headquarters | Novato, California | 60 Leveroni Court, Novato, CA 94949 |
| Founded | 2010 | Incorporated June 13, 2011 |
| CEO | Emil D. Kakkis, M.D., Ph.D. | Founder; former BioMarin CMO |
| Employees | 1,294 (Dec 2024) | 10% reduction planned 2026 |
| Market Cap | ~$2.1 billion | March 2026 (down from $3.89B in Dec 2024) |
| Line Item | FY 2024 | FY 2023 | YoY Change |
|---|---|---|---|
| Total Revenue | $560.2M | $434.0M | +29% |
| Crysvita Revenue | $410M | $328M (est.) | +25% |
| Dojolvi Revenue | $88M | $70M (est.) | +25% |
| Operating Expenses | $1,096M | ~$1,050M (est.) | +4% |
| R&D Expenses | $697.9M | ~$650M (est.) | +7% |
| SG&A Expenses | $321.6M | ~$310M (est.) | +4% |
| Net Loss | -$569M | -$607M | Improved $38M |
| Cash & Equivalents | $745M | ~$850M (est.) | -$105M burn |
| Product | Indication | 2024 Revenue | Growth | Peak Sales Potential |
|---|---|---|---|---|
| Crysvita (burosumab) | X-linked hypophosphatemia (XLH) | $410M | +25% YoY | $2.8B by 2033 (global) |
| Dojolvi (triheptanoin) | Long-chain fatty acid oxidation disorders (LC-FAOD) | $88M | +25% YoY | $200-300M (niche ultra-rare) |
| Mepsevii (vestronidase alfa) | Mucopolysaccharidosis VII (MPS VII) | ~$25M (est.) | +23% (Q4 YoY) | $40-60M (ultra-rare: ~200 global patients) |
| Evkeeza (evinacumab) | Homozygous familial hypercholesterolemia (HoFH) | Co-promoted (Regeneron lead) | +29% (2025 collaboration sales) | $750M-2.1B (collaboration-wide) |
| Program | Indication | Stage | Key Milestone | Peak Sales Estimate |
|---|---|---|---|---|
| DTX401 | Glycogen Storage Disease Type Ia (GSD1a) | BLA Priority Review | PDUFA: Aug 23, 2026 | $500M-1.5B |
| UX111 | MPS IIIA (Sanfilippo Type A) | BLA Resubmitted | FDA Decision: Q3 2026 | $300M-1B |
| GTX-102 | Angelman Syndrome | Phase 3 | Data Expected: H2 2026 | $900M-3B (blockbuster potential) |
| Setrusumab (UX143) | Osteogenesis Imperfecta (OI) | Phase 2/3 | Data announced 2025; path under review | $500M-1B (if approved) |
| DTX301 | Ornithine Transcarbamylase (OTC) Deficiency | Phase 3 | Enh3ance study ongoing | $500M-1.5B |
| UX701 | Wilson Disease | Phase 1/2/3 | Cyprus²+ Stage 1 positive interim data | $1B-3B |
| Region | Revenue (Est.) | % of Total | Key Markets |
|---|---|---|---|
| North America | $370-400M | 66-71% | United States, Canada |
| Europe | $110-130M | 20-23% | Germany, France, UK, Spain, Italy, Switzerland |
| Latin America | $40-60M | 7-11% | Brazil, Argentina, Mexico, Colombia |
| Japan/Asia-Pacific | $10-20M | 2-4% | Japan, Australia |
Note: Geographic revenue not officially reported by Ultragenyx in 10-K filings; estimates derived from quarterly business metrics.
| Drug | Fit Score | Undiagnosed Pool (US) | Diagnostic Delay | Ada Revenue Potential (Annual) |
|---|---|---|---|---|
| GTX-102 (Angelman) | 9/10 | 800-1,500 Ada-addressable | 2-5 years | $3.98M-13.25M |
| Crysvita (XLH) | 8/10 | 3,000-4,000 Ada-addressable | 2.7-4.1 years | $7.0M-21M |
| DTX401 (GSD1a) | 7/10 | 30-80 Ada-addressable | 3-12 months | $1.15M-4.6M |
| Evkeeza (HoFH) | 6/10 | 300-400 Ada-addressable | 5-15 years | $0.43M-1.28M |
| Dojolvi (LC-FAOD) | 5/10 | 400-500 Ada-addressable | Minimal (NBS era) | $0.25M-0.75M |
| Mepsevii (MPS VII) | 2/10 | 3-8 Ada-addressable | 6 months-3 years | $0.007M-0.018M |
Recommendation: Tier 1 priorities are GTX-102 (Angelman Syndrome) and Crysvita (XLH), offering the largest undiagnosed pools, substantial diagnostic delays (2-5 years), high per-patient lifetime value, and distinctive symptom patterns Ada can surface. GTX-102 partnership contingent on Phase 3 success (H2 2026 data).
| Company | Market Cap (Approx.) | Key Focus |
|---|---|---|
| BioMarin (BMRN) | ~$10-15B | Enzyme replacement, gene therapy (hemophilia A); MPS, metabolic |
| Sarepta (SRPT) | ~$5-8B | Neuromuscular (DMD); ASO and gene therapy platforms |
| Alnylam (ALNY) | ~$20-25B | RNAi platform; metabolic, hematologic rare diseases |
| Ultragenyx (RARE) | ~$2.0-2.2B | Rare/ultra-rare genetic diseases; gene therapy, ASO, mAb |
| Catalyst | Timing | Impact |
|---|---|---|
| DTX401 FDA Decision | Aug 23, 2026 (PDUFA) | Approval: $500M-1.5B peak sales; first commercial gene therapy |
| UX111 FDA Decision | Q3 2026 | Approval: $300M-1B peak sales; accelerated pathway |
| GTX-102 Phase 3 Data | H2 2026 | Positive: BLA 2027; blockbuster potential ($900M-3B) |
| 2026 Revenue Guidance | Full Year | $730-760M (8-13% growth); Crysvita $500-520M, Dojolvi $100-110M |
| Profitability Target | 2027 | Break-even to positive EBITDA via cost cuts + new launches |
Price Target: $77.48 consensus (range $25-$140; median $50.50)
Ratings: 19 Buy, 1 Hold, 0 Sell (out of 36 analysts)
Upside: 114% from current $36.30 to consensus $77.48
This deep dive cites 130+ sources across regulatory filings, earnings reports, clinical trial databases, analyst coverage, and peer-reviewed literature. Full citations available in the markdown report.