RARE on Nasdaq
Ultragenyx Pharmaceutical Inc.
In its 2026 annual report the company says: “We are a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. We have built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.”4 It is headquartered in Novato, California.1
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Last updated Oct 21, 2026.
Overview
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- TickerRARE on Nasdaq1
- IndustryPharmaceutical Preparations1
- HeadquartersNovato, California1
- Incorporated inDE2
- Business address60 Leveroni Court, Novato, Ca 949492
- Filer categoryLarge accelerated filer2
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We are a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. We have built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.4 From its latest annual report
Financials
From the company’s reports to the SEC. Every number links to the filing it came from.
Key numbers, fiscal 2025
What sells
Revenue by product, business segment and region, as the company reports it to the SEC.
Products and services
Product brought in 55% of the revenue Ultragenyx Pharmaceutical Inc. broke down this way in fiscal 2025.
Products and services by year
| Products and services | 2023 | 2024 | 2025 |
|---|---|---|---|
| Product | $180.0M | $285.0M | $369.0M |
| Royalty | $183.0M | $275.0M | $304.0M |
Regions
North America brought in 56% of the revenue Ultragenyx Pharmaceutical Inc. broke down this way in fiscal 2025.
Regions by year
| Regions | 2023 | 2024 | 2025 |
|---|---|---|---|
| North America | $307.0M | $340.0M | $377.0M |
| Latin America | $77.0M | $131.0M | $170.0M |
| EMEA | $48.0M | $80.0M | $108.0M |
| Asia Pacific | $2.0M | $9.0M | $18.0M |
Where the money goes
Where each dollar of revenue went, fiscal 2025
- Research and development68¢$750.0M
- Selling, general and admin32¢$349.0M
- Income tax0¢$4.0M
Out of $673.0M in revenue.5 The company lost money this year.
Financial statements
Every line of the income statement as filed: the last 3 fiscal years, latest first.5
| Period ending | 2025-12-31 | 2024-12-31 | 2023-12-31 |
|---|---|---|---|
| Total revenues | $673.0M | $560.0M | $434.0M |
| Cost of sales | $109.0M | $77.0M | $45.0M |
| Research and development | $750.0M | $698.0M | $648.0M |
| Selling, general and administrative | $349.0M | $321.0M | $310.0M |
| Total operating expenses | $1.2B | $1.1B | $1.0B |
| Loss from operations | -$535.0M | -$536.0M | -$569.0M |
| Interest income | $25.0M | $37.0M | $27.0M |
| Non-cash interest expense on liabilities for sales of future royalties | -$62.0M | -$63.0M | -$66.0M |
| Other income (expense) | $1.0M | -$5.0M | -$1.0M |
| Loss before income taxes | -$571.0M | -$567.0M | -$609.0M |
| (Provision for) benefit from income taxes | -$4.0M | -$2.0M | $2.0M |
| Net loss | -$575.0M | -$569.0M | -$607.0M |
| Net loss per share, basic | -$5.83 | -$6.29 | -$8.25 |
| Net loss per share, diluted | -$5.83 | -$6.29 | -$8.25 |
| Shares used in computing net loss per share, Basic | 98.6M shares | 90.5M shares | 73.5M shares |
| Shares used in computing net loss per share, Diluted | 98.6M shares | 90.5M shares | 73.5M shares |
No quarter-by-quarter statement was filed.
Every line of the balance sheet as filed: the last 4 fiscal years (annual) or 4 quarters (quarterly), latest first.5
| Period ending | 2025-12-31 | 2024-12-31 | 2023-12-31 | 2022-12-31 |
|---|---|---|---|---|
| Cash and cash equivalents | $421.0M | $174.0M | $214.0M | |
| Marketable securities | $259.0M | $436.0M | ||
| Accounts receivable, net | $158.0M | $122.0M | ||
| Inventory | $52.0M | $45.0M | ||
| Prepaid expenses and other assets | $61.0M | $40.0M | ||
| Total current assets | $951.0M | $817.0M | ||
| Property, plant, and equipment, net | $244.0M | $266.0M | ||
| Marketable securities | $57.0M | $135.0M | ||
| Intangible assets, net | $176.0M | $178.0M | ||
| Goodwill | $44.0M | $44.0M | ||
| Other assets | $60.0M | $63.0M | ||
| Total assets | $1.5B | $1.5B | ||
| Accounts payable | $31.0M | $39.0M | ||
| Accrued liabilities | $265.0M | $241.0M | ||
| Lease liabilities | $12.0M | $10.0M | ||
| Liabilities for sales of future royalties | $69.0M | $50.0M | ||
| Other liabilities | $7.0M | $4.0M | ||
| Total current liabilities | $384.0M | $344.0M | ||
| Lease liabilities | $24.0M | $30.0M | ||
| Deferred tax liabilities | $30.0M | $30.0M | ||
| Liabilities for sales of future royalties | $1.1B | $820.0M | ||
| Other liabilities | $20.0M | $17.0M | ||
| Total liabilities | $1.6B | $1.2B | ||
| Noncontrolling interest | $7.0M | $7.0M | $0 | |
| Preferred stock, par value of $0.001 per share-25.0 shares authorized; nil outstanding in 2025 and in 2024 | $0 | $0 | ||
| Common stock, par value of $0.001 per share-250.0 shares authorized; outstanding-96.6 in 2025 and 92.5 in 2024 | $0 | $0 | ||
| Treasury stock, at cost, 0.2 shares in 2025 and 0.1 shares in 2024 | -$8.0M | -$4.0M | ||
| Deferred compensation obligation | $8.0M | $4.0M | ||
| Additional paid-in capital | $4.5B | $4.2B | ||
| Accumulated other comprehensive income (loss) | $1.0M | -$1.0M | ||
| Accumulated deficit | -$4.5B | -$4.0B | ||
| Total stockholders' equity (deficit) | -$80.0M | $255.0M | $275.0M | $352.0M |
| Total liabilities, noncontrolling interest and stockholders' equity (deficit) | $1.5B | $1.5B | ||
| Preferred stock, par value | $0.00 | $0.00 | ||
| Preferred stock, shares authorized | 25.0M shares | 25.0M shares | ||
| Preferred stock, shares outstanding | 0 shares | 0 shares | ||
| Common stock, par value | $0.00 | $0.00 | ||
| Common stock, shares authorized | 250.0M shares | 250.0M shares | ||
| Common stock, shares outstanding | 96.6M shares | 92.5M shares | ||
| Treasury stock , shares | 200.0K shares | 100.0K shares |
| Period ending | 2025-12-31 | 2024-12-31 | 2023-12-31 | 2022-12-31 |
|---|---|---|---|---|
| Cash and cash equivalents | $421.0M | $174.0M | $214.0M | |
| Marketable securities | $259.0M | $436.0M | ||
| Accounts receivable, net | $158.0M | $122.0M | ||
| Inventory | $52.0M | $45.0M | ||
| Prepaid expenses and other assets | $61.0M | $40.0M | ||
| Total current assets | $951.0M | $817.0M | ||
| Property, plant, and equipment, net | $244.0M | $266.0M | ||
| Marketable securities | $57.0M | $135.0M | ||
| Intangible assets, net | $176.0M | $178.0M | ||
| Goodwill | $44.0M | $44.0M | ||
| Other assets | $60.0M | $63.0M | ||
| Total assets | $1.5B | $1.5B | ||
| Accounts payable | $31.0M | $39.0M | ||
| Accrued liabilities | $265.0M | $241.0M | ||
| Lease liabilities | $12.0M | $10.0M | ||
| Liabilities for sales of future royalties | $69.0M | $50.0M | ||
| Other liabilities | $7.0M | $4.0M | ||
| Total current liabilities | $384.0M | $344.0M | ||
| Lease liabilities | $24.0M | $30.0M | ||
| Deferred tax liabilities | $30.0M | $30.0M | ||
| Liabilities for sales of future royalties | $1.1B | $820.0M | ||
| Other liabilities | $20.0M | $17.0M | ||
| Total liabilities | $1.6B | $1.2B | ||
| Noncontrolling interest | $7.0M | $7.0M | $0 | |
| Preferred stock, par value of $0.001 per share-25.0 shares authorized; nil outstanding in 2025 and in 2024 | $0 | $0 | ||
| Common stock, par value of $0.001 per share-250.0 shares authorized; outstanding-96.6 in 2025 and 92.5 in 2024 | $0 | $0 | ||
| Treasury stock, at cost, 0.2 shares in 2025 and 0.1 shares in 2024 | -$8.0M | -$4.0M | ||
| Deferred compensation obligation | $8.0M | $4.0M | ||
| Additional paid-in capital | $4.5B | $4.2B | ||
| Accumulated other comprehensive income (loss) | $1.0M | -$1.0M | ||
| Accumulated deficit | -$4.5B | -$4.0B | ||
| Total stockholders' equity (deficit) | -$80.0M | $255.0M | $275.0M | $352.0M |
| Total liabilities, noncontrolling interest and stockholders' equity (deficit) | $1.5B | $1.5B | ||
| Preferred stock, par value | $0.00 | $0.00 | ||
| Preferred stock, shares authorized | 25.0M shares | 25.0M shares | ||
| Preferred stock, shares outstanding | 0 shares | 0 shares | ||
| Common stock, par value | $0.00 | $0.00 | ||
| Common stock, shares authorized | 250.0M shares | 250.0M shares | ||
| Common stock, shares outstanding | 96.6M shares | 92.5M shares | ||
| Treasury stock , shares | 200.0K shares | 100.0K shares |
Cash flow is reported year to date, not quarter by quarter, so there are no separate quarterly figures.
Every line of the cash flow as filed: the last 3 fiscal years, latest first.5
| Period ending | 2025-12-31 | 2024-12-31 | 2023-12-31 |
|---|---|---|---|
| Net loss | -$575.0M | -$569.0M | -$607.0M |
| Stock-based compensation | $153.0M | $158.0M | $135.0M |
| Amortization of discount on marketable securities, net | -$4.0M | -$13.0M | -$13.0M |
| Depreciation and amortization | $35.0M | $36.0M | $26.0M |
| Non-cash royalty revenue | -$112.0M | -$101.0M | -$69.0M |
| Non-cash interest expense on liabilities for sales of future royalties | $62.0M | $63.0M | $66.0M |
| Other | -$2.0M | -$2.0M | $3.0M |
| Accounts receivable | -$31.0M | -$34.0M | -$23.0M |
| Inventory | -$6.0M | -$11.0M | -$7.0M |
| Prepaid expenses and other assets | -$10.0M | $12.0M | $15.0M |
| Accounts payable, accrued, and other liabilities | $24.0M | $47.0M | -$1.0M |
| Net cash used in operating activities | -$466.0M | -$414.0M | -$475.0M |
| Purchase of property, plant, and equipment | -$6.0M | -$7.0M | -$44.0M |
| Purchase of marketable securities | -$186.0M | -$409.0M | -$526.0M |
| Proceeds from sale of marketable securities | $0 | $3.0M | $51.0M |
| Proceeds from maturities of marketable securities | $444.0M | $410.0M | $696.0M |
| Payments for intangible asset | -$15.0M | -$13.0M | -$3.0M |
| Other | -$1.0M | -$2.0M | -$6.0M |
| Net cash provided by (used in) investing activities | $236.0M | -$18.0M | $168.0M |
| Proceeds from the sale of future royalties, net | $392.0M | $0 | $0 |
| Proceeds from the issuance of common stock and pre-funded warrants in connection with underwritten public offerings, net | $0 | $381.0M | $326.0M |
| Proceeds from the issuance of common stock in connection with at-the-market offering, net | $80.0M | $0 | $53.0M |
| Proceeds from the issuance of common stock under equity plans, net | $6.0M | $11.0M | $8.0M |
| Proceeds from issuance of equity from noncontrolling interest | $0 | $7.0M | $0 |
| Other | $0 | $0 | $1.0M |
| Net cash provided by financing activities | $478.0M | $399.0M | $388.0M |
| Effect of exchange rate changes on cash | $4.0M | -$2.0M | $0 |
| Net increase (decrease) in cash, cash equivalents, and restricted cash | $252.0M | -$35.0M | $81.0M |
| Acquired lease liabilities arising from obtaining right-of-use assets and property, plant, and equipment | $6.0M | $10.0M | $22.0M |
| Non-cash interest expense on liabilities for sales of future royalties capitalized during the year into ending property, plant and equipment | $0 | $0 | $9.0M |
| Costs of intangible assets included in accounts payable, accrued and other liabilities | $5.0M | $15.0M | $10.0M |
No quarter-by-quarter statement was filed.
Corporate structure
Ultragenyx Pharmaceutical Inc. sits at the top of its group. 17 entities, 13 countries on record. Switch between the flow chart and the network, and select any entity to see its details.
Major holders of Ultragenyx Pharmaceutical Inc.
- RTW INVESTMENTS, LP Filed SCHEDULE 13G on Ultragenyx Pharmaceutical Inc., latest Aug 14, 2026 (4 filings since Feb 14, 2023)
- AQR CAPITAL MANAGEMENT LLC Filed SCHEDULE 13G on Ultragenyx Pharmaceutical Inc., latest Aug 13, 2026
- State Street Corporation Filed SCHEDULE 13G on Ultragenyx Pharmaceutical Inc., latest Aug 7, 2026 (2 filings since May 12, 2026)
- VANGUARD CAPITAL MANAGEMENT LLC Filed SCHEDULE 13G on Ultragenyx Pharmaceutical Inc., latest Jul 31, 2026
- VANGUARD PORTFOLIO MANAGEMENT LLC Filed SCHEDULE 13G/A on Ultragenyx Pharmaceutical Inc., latest Jul 31, 2026 (2 filings since Apr 29, 2026)
- BlackRock Filed SCHEDULE 13G/A on Ultragenyx Pharmaceutical Inc., latest Jul 30, 2026 (6 filings since Feb 3, 2022)
- VANGUARD GROUP INC Filed SCHEDULE 13G/A on Ultragenyx Pharmaceutical Inc., latest Mar 27, 2026 (5 filings since Feb 10, 2022)
- FMR LLC Filed SCHEDULE 13G/A on Ultragenyx Pharmaceutical Inc., latest Feb 5, 2026 (2 filings since Nov 5, 2025)
- SANDS CAPITAL MANAGEMENT, LLC Filed SCHEDULE 13G/A on Ultragenyx Pharmaceutical Inc., latest Feb 12, 2025 (2 filings since Feb 13, 2024)
- Wellington Management Company Filed SCHEDULE 13G/A on Ultragenyx Pharmaceutical Inc., latest Feb 10, 2025 (3 filings since Feb 6, 2023)
- PRICE T ROWE ASSOCIATES INC /MD/ Filed SC 13G/A on Ultragenyx Pharmaceutical Inc., latest Feb 14, 2024 (4 filings since Feb 14, 2022)
- Federated Hermes Filed SC 13G/A on Ultragenyx Pharmaceutical Inc., latest Jan 10, 2023 (2 filings since Feb 14, 2022)
- Ultragenyx Pharmaceutical Inc. Novato, California
- Arcturus Therapeutics Stake · San Diego
- Amlogenyx Inc. Subsidiary · Delaware, United States
- Rare Delaware Inc. Subsidiary · Delaware, United States
- Ultragenyx Argentina SRL Subsidiary · Argentina
- Ultragenyx Brasil Farmacêutica Ltda Subsidiary · Brazil
- Ultragenyx Canada Inc. Subsidiary · Canada
- Ultragenyx Chile Limitada Subsidiary · Chile
- Ultragenyx Colombia SAS Subsidiary · Colombia
- Ultragenyx Europe GmbH Subsidiary · Switzerland
- Ultragenyx France SAS Subsidiary · France
- Ultragenyx Germany GmbH Subsidiary · Germany
- Ultragenyx Holdco LLC Subsidiary · Delaware, United States
- Ultragenyx Japan K.K. Subsidiary · Japan
- Ultragenyx México, S. de R.L. de C.V. Subsidiary · Mexico
- Ultragenyx Netherlands B.V. Subsidiary · Netherlands
- Ultragenyx UK Ltd Subsidiary · United Kingdom
Built from subsidiaries listed in Exhibit 21 of annual reports filed with the SEC; major holders from Schedules 13D and 13G; stakes of 5% or more it reported on Schedules 13D and 13G (1 companies). Each entity in the chart links to its source.
What it owns
What Ultragenyx Pharmaceutical Inc. reports owning in its annual reports to the SEC: buildings, land and equipment, real estate and locations, investments, intangible assets and inventory, at the end of its latest fiscal year and a year before.
Property and equipment
| Item | Latest | A year before | Change |
|---|---|---|---|
| Property and equipment, before depreciation | $380.0M5 Dec 31, 2025 | $378.0M5 Dec 31, 2024 | +0.5% |
| Accumulated depreciation | $136.0M5 Dec 31, 2025 | $112.0M5 Dec 31, 2024 | +21.4% |
| Property and equipment, net | $244.0M5 Dec 31, 2025 | $266.0M5 Dec 31, 2024 | −8.3% |
| Operating-lease assets (leased space) | $23.0M5 Dec 31, 2025 | $26.0M5 Dec 31, 2024 | −11.5% |
Investments
| Item | Latest | A year before | Change |
|---|---|---|---|
| Cash and cash equivalents | $421.0M5 Dec 31, 2025 | $174.0M5 Dec 31, 2024 | +142.0% |
| Marketable securities, current | $259.0M5 Dec 31, 2025 | $436.0M5 Dec 31, 2024 | −40.6% |
| Marketable securities, non-current | $57.0M5 Dec 31, 2025 | $135.0M5 Dec 31, 2024 | −57.8% |
| Debt securities available for sale | $665.0M5 Dec 31, 2025 | $696.0M5 Dec 31, 2024 | −4.5% |
Intangibles and goodwill
| Item | Latest | A year before | Change |
|---|---|---|---|
| Goodwill | $44.0M5 Dec 31, 2025 | $44.0M5 Dec 31, 2024 | 0% |
| Intangible assets, net (excluding goodwill) | $176.0M5 Dec 31, 2025 | $178.0M5 Dec 31, 2024 | −1.1% |
| Accumulated amortization | $21.0M5 Dec 31, 2025 | $13.0M5 Dec 31, 2024 | +61.5% |
| Finite-lived intangibles, net | $47.0K5 Dec 31, 2025 | $49.3M9 Dec 31, 2024 | −99.9% |
Inventory
| Item | Latest | A year before | Change |
|---|---|---|---|
| Inventory, total | $52.0M5 Dec 31, 2025 | $45.0M5 Dec 31, 2024 | +15.6% |
| Work in process | $27.0M5 Dec 31, 2025 | $22.0M5 Dec 31, 2024 | +22.7% |
| Finished goods | $25.0M5 Dec 31, 2025 | $23.0M5 Dec 31, 2024 | +8.7% |
Properties, in its own words
Our primary operations are conducted at the leased facilities summarized in the table below. We believe our facilities are adequate and suitable for our current needs and that we will be able to obtain new or additional leased space in the future when necessary.
Property Location · Use · Lease Expiration Date
The rest of Item 2
Novato, California · Headquarters and office · December 2026
Novato, California · Laboratory and office · October 2028
Brisbane, California · Office · June 2026
Somerville, Massachusetts · Laboratory and office · January 2030
Woburn, Massachusetts · Laboratory and office · April 2028
Woburn, Massachusetts · Laboratory and office · October 2031
Bedford, Massachusetts · Manufacturing facility · Owned property
Item 2 of the 10-K filed Feb 18, 2026.4
Leadership
Who has led Ultragenyx Pharmaceutical Inc., and when. Pick a name to see everywhere that person has worked.
Chief executive
President
Chief financial officer
Chief operating officer
201520202025
- Chief executive officerEmil D. Kakkis Feb 7, 2014 to now13
- PresidentEmil D. Kakkis Feb 7, 2014 to now13
- Chief financial officerHoward Horn Oct 10, 2023 to now14
- Chief financial officerMardi Dier Oct 13, 2020 to Oct 17, 202215
- Chief financial officerShalini Sharp Feb 3, 2014 to Aug 19, 202016
- Chief operating officerWladimir Hogenhuis Oct 5, 2018 to Oct 7, 201917
Board members and other officers (24)
- EVP and Chief Medical OfficerEric Crombez May 2, 2023 to now18
- EVP & Chief Commercial OfficerErik Harris Jun 21, 2019 to now19
- EVP and Chief Medical OfficerCamille L. Bedrosian Jan 31, 2018 to Mar 3, 202320
- SVP & Chief TechOps OfficerDennis Karl Huang May 7, 2015 to now21
- OfficerJohn Richard Pinion Jul 20, 2015 to now22
- OfficerJayson Donald Alexander Dallas Aug 6, 2015 to Jun 4, 201823
- OfficerEmil D. Kakkis Jan 30, 2014 to Jan 30, 201424
- OfficerShalini Sharp Jan 30, 2014 to Oct 16, 202025
- OfficerThomas Richard Kassberg Jan 30, 2014 to Mar 4, 202526
- Controller and PAOTheodore Alan Huizenga Mar 28, 2014 to now27
- Chief Medical OfficerSunil Agarwal Aug 21, 2014 to Aug 23, 201628
- Board memberAmrit Ray Apr 22, 2022 to now29
- Board memberCorazon (corsee) D. Sanders Jun 30, 2021 to now30
- Board memberShehnaaz Suliman Feb 1, 2019 to now31
- Board memberDeborah Dunsire Apr 10, 2017 to now32
- Board memberLars Ekman Mar 21, 2016 to Jun 28, 202233
- Board memberMichael Narachi Feb 24, 2015 to now34
- Board memberDaniel G. Welch Apr 10, 2015 to now35
- Board memberClay B. Siegall Jan 30, 2014 to Aug 11, 202036
- Board memberEmil D. Kakkis Jan 30, 2014 to now24
- Board memberEran Nadav Jan 30, 2014 to Jul 17, 201437
- Board memberMarten Steen Jan 30, 2014 to Feb 3, 201438
- Board memberWilliam Aliski Jan 30, 2014 to Jun 28, 202139
- General counselKarah Herdman Parschauer Jun 22, 2016 to now40
Subsidiaries and ownership
Subsidiaries (Exhibit 21)
- Amlogenyx Inc. Delaware3
- Rare Delaware Inc. Delaware3
- Ultragenyx Argentina SRL Argentina3
- Ultragenyx Brasil Farmacêutica Ltda Brazil3
- Ultragenyx Canada Inc. Canada3
- Ultragenyx Chile Limitada Chile3
- Ultragenyx Colombia SAS Colombia3
- Ultragenyx Europe GmbH Switzerland3
- Ultragenyx France SAS France3
- Ultragenyx Germany GmbH Germany3
- Ultragenyx Holdco LLC Delaware3
- Ultragenyx Japan K.K. Japan3
- Ultragenyx México, S. de R.L. de C.V. Mexico3
- Ultragenyx Netherlands B.V. Netherlands3
- Ultragenyx UK Ltd United Kingdom3
The subsidiaries the company lists in Exhibit 21 of its annual report, as filed.
Timeline
- Oct 14, 20268-K Entered a material agreement41
- Oct 7, 20268-K Entered a material agreement42
- Sep 17, 20268-K Other events43
- Sep 2, 20268-K Other events44
- Aug 19, 20268-K Other events45
- Aug 11, 20268-K Other events46
- Aug 4, 20268-K Results of operations47
- May 18, 20268-K Executive or director change; Shareholder vote48
- May 5, 20268-K Results of operations49
- Apr 2, 20268-K Other events50
- Mar 30, 20268-K Other events51
- Mar 12, 20268-K Other events52
- Feb 7, 2014New chief executive Emil D. Kakkis becomes CEO13
Calendar
Coming up
- Nov 3, 2026Reports results for the quarter ending Sep/2026 A year earlier it reported ($1.81) a share, on Nov 4, 2025.53
When it reported results
- Aug 4, 2026Reported quarterly or annual results47
- May 5, 2026Reported quarterly or annual results49
- Feb 12, 2026Reported quarterly or annual results54
- Jan 12, 2026Reported quarterly or annual results55
- Nov 4, 2025Reported quarterly or annual results56
- Aug 5, 2025Reported quarterly or annual results57
- May 6, 2025Reported quarterly or annual results58
- Feb 13, 2025Reported quarterly or annual results59
Upcoming dates from Nasdaq's calendar; past results from the company's 8-K filings (Item 2.02). The full markets calendar
Filings
380 filings to the SEC since October 2021: 5 annual reports, 15 quarterly reports, 81 major events, 174 insider forms, 37 large holders, 68 other. The latest 20:
- Oct 14, 20268-K EDGAR
- Oct 7, 20268-K EDGAR
- Sep 17, 20268-K EDGAR
- Sep 15, 2026144 EDGAR
- Sep 15, 20264 EDGAR
- Sep 8, 20264 EDGAR
- Sep 2, 20268-K EDGAR
- Aug 19, 20268-K EDGAR
- Aug 14, 2026SCHEDULE 13G EDGAR
- Aug 13, 2026SCHEDULE 13G EDGAR
- Aug 11, 20268-K EDGAR
- Aug 7, 2026SCHEDULE 13G EDGAR
- Aug 5, 202610-Q EDGAR
- Aug 4, 20268-K EDGAR
- Aug 4, 20264 EDGAR
- Aug 4, 20264 EDGAR
- Aug 3, 2026144 EDGAR
- Jul 31, 2026SCHEDULE 13G/A EDGAR
- Jul 31, 2026SCHEDULE 13G EDGAR
- Jul 30, 2026SCHEDULE 13G/A EDGAR
The 380 filings since October 2021 can be filtered by form type and year here when JavaScript is on.
Every filing from the last five years, summarized. The full record is on SEC EDGAR.
Insider trades
109 open-market trades by officers, directors and large owners since October 2021: 0 buys and 109 sales. Grants, option exercises and gifts are left out.
| Date | Who | Trade | Shares | Price | Value | Filing |
|---|---|---|---|---|---|---|
| Sep 15, 2026 | Huizenga Theodore AlanSVP, Chief Accounting Officer | Sale | 1,283 | $13.57 | $17.4K | Form 4 |
| Sep 3, 2026 | Huizenga Theodore AlanSVP, Chief Accounting Officer | Sale | 161 | $13.99 | $2.3K | Form 4 |
| Aug 3, 2026 | Parschauer Karah HerdmanEVP and Chief Legal Officer | Sale | 1,900 | $24.92 | $47.3K | Form 4 |
| Aug 3, 2026 | Horn HowardChief Financial Officer | Sale | 4,696 | $24.92 | $117.0K | Form 4 |
| Jul 1, 2026 | Horn HowardChief Financial Officer | Sale | 4,698 | $33.13 | $155.6K | Form 4 |
| Jun 15, 2026 | Sanders Corazon (Corsee) D.Director | Sale | 2,000 | $25.05 | $50.1K | Form 4 |
| Jun 15, 2026 | Parschauer Karah HerdmanEVP and Chief Legal Officer | Sale | 1,899 | $24.62 | $46.8K | Form 4 |
| Jun 1, 2026 | Horn HowardChief Financial Officer | Sale | 4,653 | $23.77 | $110.6K | Form 4 |
| May 18, 2026 | SULIMAN SHEHNAAZDirector | Sale | 5,740 | $25.12 | $144.2K | Form 4 |
| May 5, 2026 | Crombez EricEVP and Chief Medical Officer | Sale | 344 | $24.96 | $8.6K | Form 4 |
| May 1, 2026 | Horn HowardChief Financial Officer | Sale | 4,683 | $24.90 | $116.6K | Form 4 |
| Apr 1, 2026 | Horn HowardChief Financial Officer | Sale | 4,683 | $20.97 | $98.2K | Form 4 |
| Mar 10, 2026 | Parschauer Karah HerdmanEVP and Chief Legal Officer | Sale | 3,508 | $22.67 | $79.5K | Form 4 |
| Mar 6, 2026 | Parschauer Karah HerdmanEVP and Chief Legal Officer | Sale | 6,018 | $21.40 | $128.8K | Form 4 |
| Mar 3, 2026 | Horn HowardChief Financial Officer | Sale | 1,635 | $22.40 | $36.6K | Form 4 |
| Mar 2, 2026 | Pinion John RichardSee Remarks | Sale | 10,029 | $22.80 | $228.7K | Form 4 |
| Mar 2, 2026 | Harris ErikEVP & Chief Commercial Officer | Sale | 10,539 | $22.80 | $240.3K | Form 4 |
| Mar 2, 2026 | KAKKIS EMIL DPresident & CEO | Sale | 54,404 | $22.80 | $1.2M | Form 4 |
| Mar 2, 2026 | Parschauer Karah HerdmanEVP and Chief Legal Officer | Sale | 8,135 | $22.80 | $185.5K | Form 4 |
| Mar 2, 2026 | Huizenga Theodore AlanSVP, Chief Accounting Officer | Sale | 1,632 | $22.80 | $37.2K | Form 4 |
All 109 trades can be filtered by person here when JavaScript is on.
From Forms 4 filed with the SEC.
Major holders
Investors who own more than 5% of the shares must tell the SEC: a 13D if they may seek to influence the company, a 13G if they hold passively.
- RTW INVESTMENTS, LPSCHEDULE 13G, 4 filings Feb 14, 2023 to Aug 14, 202660
- AQR CAPITAL MANAGEMENT LLCSCHEDULE 13G, 1 filing Aug 13, 202661
- State Street CorporationSCHEDULE 13G, 2 filings May 12, 2026 to Aug 7, 202662
- VANGUARD CAPITAL MANAGEMENT LLCSCHEDULE 13G, 1 filing Jul 31, 202663
- VANGUARD PORTFOLIO MANAGEMENT LLCSCHEDULE 13G/A, 2 filings Apr 29, 2026 to Jul 31, 202664
- BlackRockSCHEDULE 13G/A, 6 filings Feb 3, 2022 to Jul 30, 202665
- VANGUARD GROUP INCSCHEDULE 13G/A, 5 filings Feb 10, 2022 to Mar 27, 202666
- FMR LLCSCHEDULE 13G/A, 2 filings Nov 5, 2025 to Feb 5, 202667
- SANDS CAPITAL MANAGEMENT, LLCSCHEDULE 13G/A, 2 filings Feb 13, 2024 to Feb 12, 202568
- Wellington Management CompanySCHEDULE 13G/A, 3 filings Feb 6, 2023 to Feb 10, 202569
- PRICE T ROWE ASSOCIATES INC /MD/SC 13G/A, 4 filings Feb 14, 2022 to Feb 14, 202470
- Federated HermesSC 13G/A, 2 filings Feb 14, 2022 to Jan 10, 202371
- Capital International InvestorsSC 13G/A, 2 filings Feb 11, 2022 to Dec 9, 202272
- Capital Research Global InvestorsSC 13G/A, 1 filing Oct 8, 202173
Stakes it holds
Ultragenyx Pharmaceutical Inc. has told the SEC it owns more than 5% of 1 company, latest first (Schedules 13D and 13G).
- Arcturus TherapeuticsSC 13D/A Oct 26, 202174
Executive pay
What the company paid its top executives, from the summary compensation table in its proxy statement. Total pay includes salary, bonus, stock and option awards and other pay, as the company valued them.
| Year | Executive | Age | Salary | Total pay |
|---|---|---|---|---|
| 2025 | Emil D. Kakkis, M.D., Ph.D.President and Chief Executive Officer | 65 | $861.1K | $11.4M75 |
| 2025 | Erik HarrisChief Commercial Officer and Executive Vice President | 56 | $619.7K | $4.0M75 |
| 2025 | Eric CrombezM.D. | 53 | $630.2K | $4.0M75 |
| 2025 | Howard HornChief Financial Officer, Corporate Strategy and Executive Vice President | 48 | $604.4K | $3.8M75 |
| 2025 | Karah ParschauerChief Legal Officer and Corporate Affairs, Executive Vice President | 48 | $585.3K | $3.5M75 |
| 2024 | Emil D. Kakkis, M.D., Ph.D.President and Chief Executive Officer | 65 | $856.0K | $13.7M75 |
| 2024 | Chief Medical Officer and Executive Vice President | $591.8K | $4.3M75 | |
| 2024 | Howard HornChief Financial Officer, Corporate Strategy and Executive Vice President | 48 | $590.0K | $4.3M75 |
| 2024 | Erik HarrisChief Commercial Officer and Executive Vice President | 56 | $604.2K | $4.3M75 |
| 2023 | Emil D. Kakkis, M.D., Ph.D.President and Chief Executive Officer | 65 | $823.7K | $13.1M75 |
| 2023 | Howard HornChief Financial Officer, Corporate Strategy and Executive Vice President | 48 | $124.8K | $5.9M75 |
| 2023 | Erik HarrisChief Commercial Officer and Executive Vice President | 56 | $586.0K | $4.1M75 |
Held by these funds
The largest holders among investment managers' 13F reports for the quarter ending Jun 30, 2026, the top 25 of 100. Change is in shares since the quarter before.
| Fund | Shares | Value | Share of the fund | Change |
|---|---|---|---|---|
| BlackRock, Inc. | 10,654,276 | $355.7M | 0.0% | +3,354,678 |
| Rtw Investments, LP | 7,796,895 | $260.3M | 2.2% | +4,148,100 |
| Vanguard Portfolio Management LLC | 6,790,135 | $226.7M | 0.0% | +1,404,143 |
| Aqr Capital Management LLC | 6,560,809 | $214.1M | 0.1% | +1,982,785 |
| State Street Corp | 6,158,766 | $205.6M | 0.0% | +1,137,325 |
| Vanguard Capital Management LLC | 4,296,491 | $143.5M | 0.0% | +106,660 |
| Two Sigma Investments, LP | 3,621,200 | $120.9M | 0.1% | +1,415,380 |
| Sands Capital Management, LLC | 3,158,368 | $105.5M | 0.4% | −56,165 |
| Jacobs Levy Equity Management, Inc | 2,939,719 | $98.2M | 0.4% | +1,508,648 |
| Frontier Capital Management Co LLC | 2,847,936 | $95.1M | 0.9% | +656,754 |
| Geode Capital Management, LLC | 2,578,828 | $86.1M | 0.0% | +729,437 |
| Bellevue Group AG | 1,982,775 | $66.2M | 1.4% | −397,225 |
| D. E. Shaw & Co., Inc. | 1,730,193 | $57.8M | 0.0% | +469,175 |
| Sofinnova Investments, Inc. | 1,717,505 | $57.3M | 3.1% | −1,428,383 |
| Adage Capital Partners Gp, L.l.c. | 1,580,000 | $52.8M | 0.1% | +1,100,000 |
| Mpm Bioimpact LLC | 1,418,215 | $47.4M | 2.9% | New |
| Morgan Stanley | 1,324,805 | $44.2M | 0.0% | −175,723 |
| Wellington Management Group Llp | 1,250,464 | $41.8M | 0.0% | −1,390,348 |
| Voloridge Investment Management, LLC | 1,241,556 | $41.5M | 0.2% | −185,278 |
| Bank Of America Corp /de/ | 1,148,454 | $38.3M | 0.0% | +101,908 |
| Candriam S.C.A. | 1,001,841 | $33.4M | 0.2% | −94,341 |
| Rock Springs Capital Management LP | 967,855 | $32.3M | 1.6% | −250,918 |
| Jefferies Financial Group Inc. | 929,626 | $31.0M | 0.3% | +914,138 |
| Northern Trust Corp | 920,314 | $30.7M | 0.0% | +384,748 |
| Eagle Health Investments LP | 884,121 | $29.5M | 4.9% | No change |
13F reports cover disclosed long positions in U.S. stocks only.
Members of Congress who traded it
5 trades disclosed by members of Congress. “Vs S&P since” is the stock’s change since the trade minus the S&P 500’s.
| Date | Member | Trade | Amount | Vs S&P since | Filing |
|---|---|---|---|---|---|
| Sep 29, 2026 | Gilbert Ray Cisneros, Jr.Democratic, House | Buy | $1,001 - $15,000 | −2.0% | Report |
| Mar 3, 2026 | Gilbert Ray Cisneros, Jr.Democratic, House | Buy | $1,001 - $15,000 | −44.7% | Report |
| Feb 18, 2026 | Gilbert Ray Cisneros, Jr.Democratic, House | Buy | $1,001 - $15,000 | −38.3% | Report |
| Aug 8, 2025 | Gilbert Ray Cisneros, Jr.Democratic, House | Buy | $1,001 - $15,000 | −66.0% | Report |
| Jul 15, 2025 | Gilbert Ray Cisneros, Jr.Democratic, House | Buy | $1,001 - $15,000 | −68.3% | Report |
Members report amounts as ranges, not exact figures. Returns are price-only (no dividends).
SEC filer profile
How Ultragenyx Pharmaceutical Inc. is registered with the Securities and Exchange Commission, from its EDGAR record and the cover page of its latest annual report.
- Name on EDGARUltragenyx Pharmaceutical Inc.1
- CIK0001515673
- Employer ID (EIN)27-25460834
- Filer statusLarge accelerated filer not an emerging growth company; not a smaller reporting company; not a shell company4
- Incorporated inDelaware4
- Fiscal year endsDecember 311
- Industry code (SIC)2834 Pharmaceutical Preparations1
- SEC reviewing officeOffice of Life Sciences, Division of Corporation Finance1
- Business address60 Leveroni Court, Novato, CA 949491
- Phone415-483-88001
- Shares outstanding98,588,873 as of Jul 31, 202611
- Public float$3.4B as of Jun 30, 20254
- AuditorErnst & Young LLP, San Mateo, California PCAOB ID 42; it also attests to the company’s internal control over financial reporting4
- On EDGAR sinceMar 16, 20111
- Filings1,142 in all: 4 (574), 8-K (208), SC 13G/A (62), 144 (39), 10-Q (38), 3 (31), 424B5 (18), SC 13G (18)1
All 41 form types
S-8 (15), DEFA14A (14), 10-K (13), DEF 14A (13), 4/A (12), 5 (8), SCHEDULE 13G (7), SCHEDULE 13G/A (7), CT ORDER (6), SC TO-T/A (6), SC TO-C (5), CORRESP (4), D (4), S-1/A (4), S-3ASR (4), SC 13D/A (4), ARS (3), UPLOAD (3), 424B4 (2), 424B7 (2), EFFECT (2), FWP (2), S-1 (2), SC 13D (2), SEC STAFF LETTER (2), 10-Q/A (1), 3/A (1), 8-A12B (1), 8-K/A (1), CERTNAS (1), DRS (1), S-1MEF (1), SC TO-T (1)
- Latest filingsAnnual report (10-K) Feb 18, 2026
Quarterly report (10-Q) Aug 5, 2026
Proxy statement (DEF 14A) Mar 27, 2026
Latest 8-K (8-K) Oct 14, 2026
Securities registered for trading
| Class | Symbol | Exchange |
|---|---|---|
| Common Stock, $0.001 par value | RARE | The Nasdaq Global Select Market |
From the cover page of the 10-K filed Feb 18, 2026 (Section 12(b) of the Exchange Act).4
Risk factors and legal proceedings
The risks Ultragenyx Pharmaceutical Inc. names in Item 1A of its 10-K filed Feb 18, 2026: 63 risks under these headings, as written.4
- Because of the following factors, as well as other factors affecting our financial condition and operating results, past financial performance should not be considered to be a reliable indicator of future performance, and investors should not use historical trends to anticipate results or trends in future periods.
- Risks Related to Our Financial Condition and Capital Requirements
- We have a history of operating losses and expect to continue to incur operating losses in the near term.
- Our future financial performance depends on the successful commercialization of our products and product candidates.
- We may need to raise additional capital to fund our activities. Such additional financing may not be available on acceptable terms, if at all. Failure to obtain this necessary capital when needed may force us to delay, limit, or terminate our product development efforts or other activities.
- Risks Related to the Discovery and Development of Our Product Candidates
- Clinical drug development involves a lengthy, complex, and expensive process with uncertain outcomes and the potential for substantial delays, and the results of earlier studies may not be predictive of future study results.
- If we do not achieve our projected development goals in the time frames we announce and expect, the commercialization of our products may be delayed and the credibility of our management may be adversely affected and, as a result, our stock price may decline.
- We may find it difficult to identify and enroll patients in our clinical studies due to a variety of factors, including the limited number of patients who have the diseases for which our product candidates are being studied and other unforeseen events. Difficulty in enrolling patients could delay or prevent clinical studies of our product candidates.
- The regulatory approval processes of the FDA and comparable foreign authorities are lengthy, time consuming, and inherently unpredictable. Even if we achieve positive results in our pre-clinical and clinical studies, if we are ultimately unable to obtain timely regulatory approval for our product candidates, our business will be substantially harmed.
- Fast Track, Breakthrough Therapy, Priority Review, or Regenerative Medicine Advanced Therapy, or RMAT, designations by the FDA, or access to the Priority Medicine scheme, or PRIME, by the EMA, for our product candidates, if granted, may not lead to
- faster development or regulatory review or approval process, and it does not increase the likelihood that our product candidates will receive marketing approval.
The other 53 risks
- Our product candidates may cause undesirable side effects or have other properties that could delay or prevent their regulatory approval, limit the commercial profile of an approved label, or result in significant negative consequences following marketing approval, if any.
- Serious adverse events in clinical trials involving gene therapy product candidates may damage public perception of the safety of our product candidates, increase government regulation, and adversely affect our ability to obtain regulatory approvals for our product candidates or conduct our business.
- Gene therapy product candidates are novel, complex, expensive and difficult to manufacture. We could experience manufacturing problems that result in delays in developing and commercializing these programs or otherwise harm our business.
- Our products are subject to regulatory scrutiny, even after approval.
- Product liability lawsuits against us could cause us to incur substantial liabilities and could limit commercialization of our approved products or product candidates.
- If we are unable to identify, source, and develop effective biomarkers, or our collaborators are unable to successfully develop and commercialize companion diagnostics for our product candidates, or experience significant delays in doing so, we may not realize the full commercial potential of our product candidates.
- Risks Related to our Reliance on Third Parties
- We rely on third parties to conduct our nonclinical and clinical studies and perform other tasks for us. If these third parties do not successfully carry out their contractual duties, meet expected deadlines, or comply with regulatory requirements, we may be exposed to sub-optimal quality and reputational harm, we may not be able to obtain regulatory approval for or commercialize our product candidates, and our business could be substantially harmed.
- We are dependent on KKC for the commercialization of Crysvita in our markets, including the U.S. and Canada, and for our supply of Crysvita in our markets. Failure by KKC to commercialize Crysvita in those markets, or to supply Crysvita to us, could result in a material adverse effect on our business and operating results.
- We rely on third parties to manufacture our products and our product candidates and we are subject to a multitude of manufacturing risks, any of which could substantially increase our costs and limit the supply of our products and product candidates.
- The drug substance and drug product for our products and most of our product candidates are currently acquired from single-source suppliers. The loss of these suppliers, or their failure to supply us with the necessary drug substance or drug product, could materially and adversely affect our business.
- The actions of distributors and specialty pharmacies could affect our ability to sell or market products profitably. Fluctuations in buying or distribution patterns by such distributors and specialty pharmacies could adversely affect our revenues, financial condition, or results of operations.
- Risks Related to Commercialization of Our Products and Product Candidates
- If the market opportunities for our products and product candidates are smaller than we believe they are, our revenue may be adversely affected, and our business may suffer.
- We face intense competition and rapid technological change, including the use of artificial intelligence, or AI, and the possibility that our competitors may develop therapies that are similar, more advanced, or more effective than ours, which may adversely affect our financial condition and our ability to successfully commercialize our product candidates.
- If we are unable to expand our existing commercial infrastructure or enter into agreements with third parties to market and sell our products and product candidates, as needed, we may be unable to increase our revenue.
- The commercial success of any current or future product will depend upon the degree of market acceptance by physicians, patients, third-party payors, and others in the medical community.
- The insurance coverage and reimbursement status of newly approved products is uncertain. Failure to obtain or maintain adequate coverage and reimbursement for new or current products could limit our ability to market those products and decrease our ability to generate revenue.
- Risks Related to Our Intellectual Property
- If we are unable to obtain and maintain effective patent rights for our products, product candidates, or any future product candidates, we may not be able to compete effectively in our markets.
- We may not have sufficient patent terms to effectively protect our products and business.
- Patent law and rule changes could increase the uncertainties and costs surrounding the prosecution of our patent applications and the enforcement or defense of our issued patents.
- If we are unable to maintain effective proprietary rights for our products, product candidates, or any future product candidates, we may not be able to compete effectively in our markets.
- Claims of intellectual property infringement may prevent or delay our development and commercialization efforts.
- We may not be successful in obtaining or maintaining necessary rights to our product candidates through acquisitions and in-licenses.
- We may face competition from biosimilars, which may have a material adverse impact on the future commercial prospects of our biological products and product candidates.
- Competitors could enter the market with generic versions of Dojolvi or our small-molecule product candidates, which may result in a material decline in sales of affected products.
- The patent protection and patent prosecution for some of our products and product candidates is dependent on third parties.
- If we fail to comply with our obligations in the agreements under which we license intellectual property and other rights from third parties or otherwise experience disruptions to our business relationships with our licensors, we could lose license rights that are important to our business.
- From time to time, we are involved in lawsuits to protect or enforce our patents or the patents of our licensors, or may be subject to claims that challenge the inventorship or ownership of our patents or other intellectual property, which could be expensive, time consuming, and result in unfavorable outcomes.
- Changes to patent laws in the U.S. and other jurisdictions could diminish the value of patents in general, thereby impairing our ability to protect our products.
- We may not be able to protect our intellectual property rights throughout the world.
- Risks Related to Our Business Operations
- We have limited experience as a company operating our own manufacturing facility and may experience unexpected costs or challenges.
- Our future success depends in part on our ability to retain our Founder, President, and Chief Executive Officer and to attract, retain, and motivate other qualified personnel.
- If we fail to obtain or maintain orphan drug exclusivity for our products, our competitors may sell products to treat the same conditions and our revenue will be reduced. If another party obtains orphan drug exclusivity for a product that is essentially the same as a product we are developing for a particular indication, we may be precluded or delayed from commercializing the product in that indication.
- Our operating results would be adversely impacted if our intangible assets become impaired.
- We may not be successful in our efforts to identify, license, discover, develop, or commercialize additional product candidates.
- We may expend our limited resources to pursue a particular product, product candidate or indication and fail to capitalize on products, product candidates or indications that may be more profitable or for which there is a greater likelihood of success.
- Regulatory standards are subject to change over time, making it difficult to accurately predict the likelihood of marketing approval even when clinical trials meet their endpoints.
- Failure to comply with laws and regulations could harm our business and our reputation.
- Increasing use of social media could give rise to liability and result in harm to our business.
- International expansion of our business exposes us to business, regulatory, political, operational, financial, and economic risks associated with doing business outside of the U.S.
- The FDA and other regulatory agencies actively enforce the laws and regulations prohibiting the promotion of off-label uses. If we are found to have promoted off-label uses, we may become subject to significant liability.
- Our business and operations may be materially adversely affected in the event of computer system failures or security breaches.
- We or the third parties upon whom we depend may be adversely affected by earthquakes or other natural disasters and our business continuity and disaster recovery plans may not adequately protect us from a serious disaster.
- We may experience difficulties, delays or unexpected costs and not achieve anticipated benefits and savings from our recently initiated strategic restructuring plan.
- We may acquire companies or products or engage in strategic transactions, which could divert our management’s attention and cause us to incur various costs and expenses, or result in fluctuations with respect to the value of such investment, which could impact our operating results.
- Risks Related to Ownership of Our Common Stock
- The market price of our common stock may be highly volatile.
- Future sales and issuances of our common stock or rights to purchase common stock, including pursuant to our equity incentive plans, could result in additional dilution of the percentage ownership of our stockholders and could cause our stock price to fall.
- Provisions in our amended and restated certificate of incorporation and by-laws, as well as provisions of Delaware law, could make it more difficult for a third party to acquire us or increase the cost of acquiring us, even if doing so would benefit our stockholders, or remove our current management.
- Our amended and restated certificate of incorporation provides that the Court of Chancery of the State of Delaware is the sole and exclusive forum for substantially all disputes between us and our stockholders, which could limit our stockholders’ ability to obtain a favorable judicial forum for disputes with us or our directors, officers or employees.
- General Risk Factors
- We may incur additional tax liabilities related to our operations.
- Our ability to use our net operating loss carryforwards and certain other tax attributes may be limited.
- Litigation may substantially increase our costs and harm our business.
- Our business and operations could be negatively affected if we become subject to stockholder activism or hostile bids, which could cause us to incur significant expense, hinder execution of our business strategy and impact our stock price.
- Scrutiny regarding ESG practices and disclosures, as well as existing and proposed laws and regulations related to these topics, could result in additional costs and adversely impact our business and reputation.
Legal proceedings
Ultragenyx Pharmaceutical Inc. and Baylor Research Institute v. Navinta LLC, Aurobindo Pharma Limited, Aurobindo Pharma USA, Inc., Esjay Pharma Private Limited and Esjay Pharma LLC
In September 2024, we filed a patent infringement suit under the Hatch-Waxman Act against Navinta LLC, or Navinta, Aurobindo Pharma Limited, or Aurobindo, and Esjay Pharma LLC, or Esjay, in the United States District Court for the District of New Jersey. The suit is in response to notices from Navinta, Aurobindo, and Esjay concerning the filing of Abbreviated New Drug Applications, or ANDAs, with the FDA, seeking FDA approval to market a generic version of Dojolvi® (triheptanoin). The filing of the suit triggers a stay preventing the FDA from granting the ANDAs final approval, which stay extends to December 30, 2027 (i.e., the date that is seven and one-half years from the June 30, 2020 approval of Dojolvi). We intend to vigorously defend our intellectual property. In addition to the issued patents for Dojolvi listed in the Orange Book, in February 2026, a patent relating to certain pharmaceutical compositions of triheptanoin, including Dojolvi, issued in the United States and is expected to expire in 2034. We expect to list this patent in the Orange Book shortly. Dojolvi is also protected in the U.S. by orphan drug exclusivity for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD) until 2027.
In December 2024, Esjay filed a motion to dismiss the suit and in April 2025, Navinta filed a motion for judgment on the pleadings, which we opposed in May 2025. Discovery is ongoing.
Ultragenyx Pharmaceutical Inc. v. Catalent Maryland, Inc. and Catalent Pharma Solutions LLC
The opening of Item 3, Legal Proceedings.4
Sources
The numbers on this page point here. Each one links to the record it came from.
- 1
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- 14SEC EDGARHoward Horn, Chief financial officer
- 15SEC EDGARMardi Dier, Chief financial officer
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- 21
- 22SEC EDGARJohn Richard Pinion, Officer
- 23
- 24SEC EDGAREmil D. Kakkis, Officer
- 25SEC EDGARShalini Sharp, Officer
- 26SEC EDGARThomas Richard Kassberg, Officer
- 27
- 28SEC EDGARSunil Agarwal, Chief Medical Officer
- 29SEC EDGARAmrit Ray, Board member
- 30
- 31SEC EDGARShehnaaz Suliman, Board member
- 32SEC EDGARDeborah Dunsire, Board member
- 33SEC EDGARLars Ekman, Board member
- 34SEC EDGARMichael Narachi, Board member
- 35SEC EDGARDaniel G. Welch, Board member
- 36SEC EDGARClay B. Siegall, Board member
- 37SEC EDGAREran Nadav, Board member
- 38SEC EDGARMarten Steen, Board member
- 39SEC EDGARWilliam Aliski, Board member
- 40
- 41SEC EDGAREntered a material agreement
- 42SEC EDGAREntered a material agreement
- 43SEC EDGAROther events
- 44SEC EDGAROther events
- 45SEC EDGAROther events
- 46SEC EDGAROther events
- 47SEC EDGARResults of operations
- 48
- 49SEC EDGARResults of operations
- 50SEC EDGAROther events
- 51SEC EDGAROther events
- 52SEC EDGAROther events
- 53NasdaqNasdaq calendar
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