Last updated
Revenue
R&D Expenses
Operating CF
Total Assets
Total Liabilities
Equity
Shares Out.
D/E Ratio
Dilution Watch
Financials
52W LOW $12.7352W HIGH $39.89
Volume
2.93 M
Value Traded
62.77 M
Short % Float
18.37%
+6.76%Week
+7.5%1 Month
-53.4%3 Month
-34.49%6 Month
-79.38%5 Year
-48.93%All Time
Cash Data
- Monthly Burn
- Burn Trend
- Dilution Risk
- SEC Filing
Key Stats
Market Cap
1.53 B
EPS (TTM)
-5.94
P/E Ratio
-
Ent. Value
1.09 B
Total Shares
98.56 M
Float Shares
90.19 M
Insiders
8.52%
Institutions
105.33%
Upcoming Catalysts
FDA decisions, readouts and PDUFA dates · scored by probability of approval
PoA blends historical approval base rates, trial design score and FDA review track record.
RARE Catalyst Timeline
Dated clinical, regulatory and corporate events for Ultragenyx Pharmaceutical Inc.
Catalyst Timeline
Dated clinical, regulatory & corporate events for Ultragenyx Pharmaceutical Inc.
835Total events
11Upcoming
237Tier-1 (high impact)
2012 to 2028Coverage
Upcoming catalysts 2
Event history 50
▲PRV SoldDesignation
Ultragenyx enters agreement to sell Rare Pediatric Disease PRV for $210 million
glycogen storage disease type Ia (GSDIa)source ↗
▲FDA ApprovalFAYUVIApprovedFDA Decision
FDA grants standard full approval of FAYUVI for MPS IIIA
Sanfilippo syndrome Type A (MPS IIIA)source ↗
▲PRV GrantedFAYUVIApprovedDesignation
Ultragenyx receives Priority Review Voucher upon FAYUVI approval
Sanfilippo syndrome Type A (MPS IIIA)source ↗
●R&D DayPresentation
Ultragenyx to host conference call on September 17, 2026 at 5:30 p.m. ET
▼Primary Endpoint MissedGTX-102Clinical Data
Phase 3 Aspire did not achieve primary endpoint of change from Baseline in Bayley-4 cognitive raw score
Angelman syndromesource ↗
▼Secondary Endpoint DataGTX-102Clinical Data
Phase 3 Aspire did not achieve key secondary endpoint of net response in MDRI
Angelman syndromesource ↗
▲Full ResultsDTX401ApprovedClinical Data
Publication of 96-week randomized, placebo-controlled trial results for DTX401 in GSDIa
GSDIasource ↗
●Timing GuidanceFAYUVIApprovedPresentation
fayuvi.com expected to be live within the coming days
Sanfilippo syndrome Type A (MPS IIIA)source ↗
▲FDA ApprovalGENGLYCOSApprovedFDA Decision
FDA approval of GENGLYCOS gene therapy
Glycogen Storage Disease Type Ia (GSDIa)source ↗
▲PRV GrantedGENGLYCOSApprovedDesignation
Priority Review Voucher granted upon approval
Glycogen Storage Disease Type Ia (GSDIa)source ↗
◆Oral PresentationPresentation
Ultragenyx to participate in fireside chat at Goldman Sachs 47th Annual Global Healthcare Conference
◆Quarterly UpdateCorporate
Class action lawsuit filed; lead plaintiff application deadline April 6, 2026
▲FDA Filing AcceptedUX111ApprovedRegulatory Filing
FDA accepted resubmitted BLA for UX111
Sanfilippo syndrome type Asource ↗
▲Topline ReadoutDTX301Clinical Data
Positive 36-week topline data from Phase 3 study of DTX301
Ornithine Transcarbamylase (OTC) Deficiencysource ↗
▲Primary Endpoint MetDTX301Clinical Data
Primary endpoint of ammonia control met at Week 36
Ornithine Transcarbamylase (OTC) Deficiencysource ↗
▲Full ResultsDTX301Clinical Data
Full 36-week results including secondary endpoints and safety
Ornithine Transcarbamylase (OTC) Deficiencysource ↗
▲FDA Filing AcceptedDTX401ApprovedRegulatory Filing
FDA accepted BLA for DTX401
Glycogen Storage Disease Type Iasource ↗
▲Priority ReviewDTX401ApprovedRegulatory Filing
Priority Review granted for DTX401 BLA
Glycogen Storage Disease Type Iasource ↗
▼Restructuring LayoffsCorporate
Strategic restructuring plan initiated with 10% workforce reduction (~130 employees)
▼Complete Response LetterUX111ApprovedFDA Decision
FDA issued Incomplete Response Letter for UX111 BLA resubmission
Sanfilippo syndrome type A (MPS IIIA)source ↗
●Enrollment MilestoneUX701Trial
Enrollment complete for fourth cohort of Cyprus2+ study
Wilson diseasesource ↗
▲Enrollment CompleteGTX-102Trial
Enrollment completed in Phase 3 Aspire study
Angelman syndromesource ↗
▼Quarterly UpdateCorporate
Class action lawsuit filed against Ultragenyx; lead plaintiff deadline April 6, 2026
▲FDA Filing AcceptedDTX401ApprovedRegulatory Filing
FDA accepted BLA for DTX401 with Priority Review
Glycogen storage disease type Iasource ↗
◆Oral PresentationUX111ApprovedPresentation
Presentation of longer-term clinical data at WORLDSymposium 2026
Sanfilippo syndrome type A (MPS IIIA)source ↗
▲BLA SubmissionUX111ApprovedRegulatory Filing
Resubmitted BLA for UX111 to FDA
Sanfilippo syndrome type A (MPS IIIA)source ↗
◆Oral PresentationSetrusumabPresentation
UX143 Phase 3 Orbit and Cosmic data presented at J.P. Morgan Healthcare Conference
osteogenesis imperfectasource ↗
●BLA SubmissionUX111ApprovedRegulatory Filing
BLA resubmitted for UX111 in January 2026
Sanfilippo syndrome type A (MPS IIIA)source ↗
▲FDA ApprovalGENGLYCOSApprovedFDA Decision
Recent approval of GENGLYCOS for glycogen storage disease type Ia
glycogen storage disease type Iasource ↗
▲Quarterly UpdateCorporate
Preliminary 2025 total revenue $672M-$674M, exceeding top end of guidance
▼Primary Endpoint MissedSetrusumabClinical Data
Phase 3 Cosmic study did not achieve statistical significance against primary endpoint of reduction in annualized clinical fracture rate compared to bisphosphonates
Osteogenesis Imperfectasource ↗
▼Topline ReadoutSetrusumabClinical Data
Phase 3 Cosmic study topline results: primary endpoint not met
Osteogenesis Imperfectasource ↗
▼Primary Endpoint MissedSetrusumabClinical Data
Phase III ORBIT study failed to achieve statistically significant reduction in annualized fracture rate
Osteogenesis Imperfectasource ↗
▼Topline ReadoutSetrusumabClinical Data
Phase 3 Cosmic study topline results: failed to demonstrate statistically significant reduction in annualized fracture rates
Osteogenesis Imperfectasource ↗
▼Program DiscontinuationSetrusumabCorporate
Ultragenyx evaluating planned operations and implementing significant expense reductions after Phase 3 failures
Osteogenesis Imperfectasource ↗
▼Restructuring LayoffsCorporate
Company evaluating planned operations and implementing significant expense reductions
▼Quarterly UpdateCorporate
Company announced evaluation of planned operations and implementation of significant expense reductions
●BLA SubmissionDTX401ApprovedRegulatory Filing
BLA rolling submission for DTX401 completed
glycogen storage disease type Ia (GSDIa)source ↗
▲Rolling Submission CompletedDTX401ApprovedRegulatory Filing
BLA rolling submission completed for DTX401
glycogen storage disease type Iasource ↗
▲Enrollment CompleteUX701Trial
UX701 Cyprus2+ study fourth cohort enrollment complete
Wilson diseasesource ↗
▲Debt Royalty FinancingCorporate
Sale of additional 25% of North American Crysvita royalty interest to OMERS for $400 million
Past FDA Catalysts and PDUFA Decisions
How RARE actually traded into and out of each decision
| Date | Drug | Catalyst | Stage | Reaction | Event Move % | Best Trade % |
|---|---|---|---|---|---|---|
| 2026-09-17 | UX111 (rebisufligene etisparvovec) | PDUFA Date Approved | PDUFA |
Reaction: 30 trading days around the event · Event Move: close to close on decision day · Best Trade: peak run-up from entry 10 days before the decision
Drug Pipeline Intelligence
C41 / 100
Pipeline Score
$200M
Pipeline Value
$1.7B
Commercial Value
Fair Value
Valuation Signal
5
Drugs Scored
0.4x
rNPV / MCap
Top 38%
Micro Cap(rank 251 of 660)
Percentile Rank
Ultragenyx Pharmaceutical Inc. carries a moderate pipeline score (41/100), with $742M risk-adjusted pipeline value, led by BPS804 in Osteopenia (Phase 2).
Commercial products (revenue basis: trailing 4 quarters of total revenue (10-K and 10-Q), to Jun 2026; product lines: last four quarters to Jun 2026 (10-K and 10-Q)) Revenue $717M x 2.4 = $1.7B
| Product | Revenue | Prior period | Change |
|---|---|---|---|
| Crysvita | $187M | $148M | +26.1% |
| Dojolvi | $101M | $92M | +9.4% |
| Evkeeza | $73M | $46M | +59.1% |
| Mepsevii | $37M | $34M | +8% |
| Drug | Indication | Phase | PTRS | rNPV | Status | Enroll | Velocity | Design | Est. Completion | ML Signal | Last Change |
|---|---|---|---|---|---|---|---|---|---|---|---|
| BPS804 Small moleculeCompletedNCT01406977 | Hypophosphatasia | Phase 2 | COMPLETED | 8 | Sep 1, 2012 |
Clinical Trial Results
Readouts, endpoints and source filings for every RARE program
| Drug Name | Indication | Phase | Date | Trial Results Summary | Title | Source |
|---|---|---|---|---|---|---|
| Osteogenesis Imperfecta | Phase 3 | 2026-03-30 | statistically significant reduction in annualized fracture rateRead More | RARE Investors Have Opportunity to Lead Ultragenyx Pharmaceutical Inc. Securities Fraud Lawsuit with the Schall Law FirmRead More |
Inside Trades
| Insider | Side | Shares | Price | Value | Date |
|---|---|---|---|---|---|
| Huizenga Theodore AlanOfficer (SVP, Chief Accounting Officer) | Sell | -1,283 67,863 held | $13.57 | 09/15/2026 |
Hedge Fund 13F Activity
Hedge Funds invested in RARE
| Fund | % of Portfolio | Current MV | Shares Owned | Activity |
|---|---|---|---|---|
| RTW INVESTMENTS, LP | 2.2 % (175 %) | 260.34 M | 7.80 M | 113.68% ( 4.15 M) |
RARE Institutional Ownership Trends
Options Data
Option Volume
Open Interest
Call OI
Put OI
Put-Call Ratio
OI 30-day avg
Today vs avg
Option Volume
Call Vol
Put Vol
Put-Call Ratio
Vol 30-day avg
Today vs avg
Option Chain Statistics
| Expiry | Call Vol | Put Vol | Vol P/C | Call OI | Put OI | OI P/C | IV Call | IV Call OI-Wtd | IV Call Vol-Wtd | IV Put | IV Put OI-Wtd | IV Put Vol-Wtd |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 2026-10-16 | ||||||||||||
Real-Time Option Chain
| Calls | Strike | Puts | ||||
|---|---|---|---|---|---|---|
| Last | Vol | OI | Last | Vol | OI | |
| No data available | ||||||
Calls vs Puts by Expiry
CallsPutsLog scale
Competitive positioning
How RARE ranks across every disease it competes in
Competitive Position is a premium feature
See how RARE ranks against every competitor across each disease it develops in: phase, best drug, trials and likelihood of approval.
Start free trialRARE News
RARE▲ +1.2%today
Ultragenyx Enters into Agreement to Sell Rare Pediatric Disease Priority Review Voucher for $210 Million
Ultragenyx Pharmaceutical Inc. has announced a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher for $210 million. This voucher was awarded following the FDA approval of GENGLYCOS, a treatment for glycogen storage disease type Ia. The funds will aid in advancing therapies for rare diseases, although the transaction is subject to customary closing conditions.
Read more →RAREUltragenyx Announces Approval of FAYUVI™ Gene Therapy, the First-Ever FDA-Approved Treatment for Sanfilippo Syndrome Type A (MPS IIIA)
Ultragenyx Pharmaceutical Inc. announced that the FDA has granted full approval for FAYUVI™, the first-ever treatment for Sanfilippo syndrome Type A. This gene therapy aims to halt the disease's progression and will be available to Qualified Treatment Centers within 30-60 days. The approval is a significant milestone for the affected community, offering hope to families facing this devastating condition.
Read more →RARE▼ -44%on this news
Ultragenyx Announces Phase 3 Aspire results in Angelman Syndrome
Ultragenyx announced that its Phase 3 Aspire study for apazunersen in Angelman syndrome failed to meet its primary and secondary endpoints. The results have led to disappointment from the company and the patient community. The company will evaluate the program's future and implement expense reductions while focusing on its commercial business.
Read more →RAREUltragenyx Announces the Publication of a Successful 96-Week Randomized, Placebo-Controlled Trial with Crossover Treatment of GENGLYCOS™ (also known as DTX401) AAV Gene Therapy in GSDIa in The Journal of Inherited Metabolic Disease
Ultragenyx has published results from a 96-week randomized, placebo-controlled trial of its AAV gene therapy, GENGLYCOS™ (DTX401), for GSDIa. The trial showed a mean reduction of 61% in daily cornstarch intake among participants, with many maintaining glycemic control. Notably, one-third of participants completely eliminated nighttime cornstarch dosing.
Read more →RAREUltragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
Ultragenyx Pharmaceutical Inc. has granted 45,984 restricted stock units to 23 newly hired non-executive officers as part of its Employment Inducement Plan. This grant, approved by the compensation committee, aims to attract talent in line with Nasdaq Listing Rule 5635(c)(4). The stock units will vest over four years, contingent on continued employment.
Read more →RAREUltragenyx Announces U.S. FDA Approval of GENGLYCOS™ Gene Therapy, the First-Ever FDA-Approved Treatment Designed to Treat the Underlying Cause of Glycogen Storage Disease Type Ia (GSDIa)
Ultragenyx has received FDA approval for GENGLYCOS, marking it as the first gene therapy for Glycogen Storage Disease Type Ia (GSDIa). This approval is significant as it offers a long-awaited treatment option aimed at alleviating the care burden for affected patients. Additionally, Ultragenyx has been awarded a Priority Review Voucher with this approval.
Read more →RAREUltragenyx Reports Second Quarter 2026 Financial Results and Corporate Update
Ultragenyx reported a total revenue of $214 million for the second quarter of 2026, with Crysvita® contributing $156 million and Dojolvi® $27 million. The company reaffirmed its financial guidance for 2026, projecting total revenue between $730 million and $760 million. Additionally, it expects combined R&D and SG&A expenses to remain flat or slightly decrease compared to 2025.
Read more →RAREUltragenyx to Host Conference Call for Second Quarter 2026 Financial Results and Corporate Update
Ultragenyx Pharmaceutical Inc. will host a conference call on August 4, 2026, at 5:00 p.m. ET to discuss its financial results for Q2 2026. The call will provide a corporate update and will be available for replay on the company's website for three months. Ultragenyx is dedicated to developing therapies for rare genetic diseases.
Read more →RAREUltragenyx Releases 2025 Impact Report Emphasizing Commitment to Rare Disease Patients, Innovation, and Global Impact
Ultragenyx Pharmaceutical Inc. released its 2025 Impact Report, highlighting its commitment to rare disease patients and innovation. The report details advancements in five investigational therapies and ongoing support for patients lacking access to approved treatments. Key achievements include significant investments in R&D and participation in global advocacy events, reflecting the company's dedication to improving patient outcomes.
Read more →RAREUltragenyx to Participate at Goldman Sachs 47th Annual Global Healthcare Conference
Ultragenyx Pharmaceutical Inc. will participate in the Goldman Sachs 47th Annual Global Healthcare Conference on June 9, 2026. CEO Emil Kakkis will engage in a fireside chat, which will be available via live and archived webcast. The company focuses on developing therapies for rare genetic diseases and has a strong commitment to efficient drug development.
Read more →RAREUltragenyx Reports First Quarter 2026 Financial Results and Corporate Update
Ultragenyx Pharmaceutical Inc. reported a total revenue of $136 million for Q1 2026, with significant contributions from Crysvita and Dojolvi. The company reaffirmed its financial guidance for the year, aiming for total revenue between $730 million and $760 million. Promising long-term data for GTX-102 in Angelman syndrome was highlighted, with Phase 3 results expected later this year. However, the company also reported a net loss of $185 million, reflecting increased operating expenses.
Read more →RAREUltragenyx to Participate at Bank of America’s 2026 Healthcare Conference
Ultragenyx Pharmaceutical Inc. will participate in Bank of America's 2026 Healthcare Conference on May 12, 2026. CFO Howard Horn and Chief of Staff Joshua Higa will lead a fireside chat, which will be accessible via webcast. The company focuses on developing therapies for rare genetic diseases and aims to address high unmet medical needs.
Read more →RAREUltragenyx to Host Conference Call for First Quarter 2026 Financial Results and Corporate Update
Ultragenyx Pharmaceutical Inc. will host a conference call on May 5, 2026, to discuss its financial results for Q1 2026. The call will provide insights into the company's performance and updates on its corporate strategy. Interested parties can access the live and recorded webcast through the company's website.
Read more →RAREUltragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Ultragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Read more →RAREUltragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX016, a Sialic Acid Prodrug for the Treatment of GNE Myopathy
Ultragenyx Pharmaceutical Inc. has received FDA clearance for its IND application for UX016, a prodrug designed to treat GNE myopathy. This investigational therapy aims to improve sialic acid delivery to muscle, addressing a critical need in a patient population with no approved treatments. A Phase 1/2 clinical study is set to begin in late 2026.
Read more →About Ultragenyx Pharmaceutical Inc.
Headquarters60 LEVERONI COURT, NOVATO, CA, 94949
BIOPHARMAWATCH ELITE PLUS
Trade the next RARE catalyst before the market reprices
Every FDA decision, PoA score, 13F move and dilution flag on this page, for 1,100+ biotech tickers. From $19/mo billed annually.
Cancel anytime