Ultragenyx Submits BLA for DTX401 Gene Therapy Targeting GSDIa
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Dec 30 2025
0mins
Should l Buy RARE?
Source: Newsfilter
- Significant Milestone: Ultragenyx has completed the rolling submission of its Biologics License Application (BLA) for DTX401 gene therapy, targeting approximately 6,000 GSDIa patients, marking a crucial step toward delivering the first therapy that addresses the underlying cause of the disease to the FDA.
- Clinical Data Support: The application is based on rigorous clinical data from 52 treated patients, demonstrating that DTX401 significantly reduces daily cornstarch intake while maintaining low hypoglycemia levels and improving patient-reported quality of life, indicating strong market demand and therapeutic efficacy.
- Regulatory Progress: Ultragenyx received rolling review from the FDA in August 2025 and has submitted all necessary modules, reflecting the company's strategic commitment to accelerating drug development and approval processes, which is expected to provide patients with faster access to treatment options.
- Market Potential: GSDIa is a serious metabolic disease with no approved pharmacologic therapies; the successful launch of DTX401 will fill a significant market gap, addressing the urgent need for effective treatments and further solidifying Ultragenyx's leadership in the rare disease sector.
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Analyst Views on RARE
Wall Street analysts forecast RARE stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for RARE is 61.65 USD with a low forecast of 35.00 USD and a high forecast of 120.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
18 Analyst Rating
17 Buy
1 Hold
0 Sell
Strong Buy
Current: 23.790
Low
35.00
Averages
61.65
High
120.00
Current: 23.790
Low
35.00
Averages
61.65
High
120.00
About RARE
Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company. The Company is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultrarare genetic diseases. Its therapies and clinical-stage pipeline consist of four product categories: biologics, small molecules, AAV gene therapy, and nucleic acid product candidates. Its four approved product candidates include Crysvita (burosumab) for the treatment of X-linked hypophosphatemia (XLH), and tumor-induced osteomalacia (TIO), Mepsevii (vestronidase alfa) for the treatment of mucopolysaccharidosis VII (MPSVII) or Sly Syndrome, Dojolvi (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia (HoFH). Its clinical product candidates include DTX401, DTX301, UX701, UX143, UX111, and GTX-102. UX143 for the treatment of Osteogenesis Imperfecta.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
Ultragenyx Resubmits BLA for Accelerated Approval of UX111 Gene Therapy
- Application Progress: On January 30, Ultragenyx Pharmaceutical resubmitted its Biologics License Application (BLA) to the FDA for accelerated approval of UX111 AAV9 gene therapy for Sanfilippo syndrome type A, with a review expected in Q3; if approved, it will be the first therapy for this condition.
- Market Analyst Support: Morgan Stanley analyst Maxwell Skor maintained a Buy rating on Ultragenyx with a price target of $50, reflecting confidence in the company's growth potential, while Leerink Partners also issued a Buy rating, indicating positive investor sentiment.
- Diverse Product Line: Ultragenyx focuses on therapies for rare and ultra-rare genetic diseases, with products like Crysvita, Mepsevii, Dojolvi, and Evkeeza, showcasing its broad market presence and potential in the biopharmaceutical sector.
- Investment Outlook Assessment: While RARE is viewed as a promising investment, analysts suggest that certain AI stocks may offer greater upside potential and lower downside risk, urging investors to carefully evaluate market dynamics to optimize their portfolios.
See More
Ultragenyx Pharmaceutical Class Action Lawsuit Announced
- Class Action Initiation: Rosen Law Firm has announced a class action lawsuit on behalf of investors who purchased Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) common stock between August 3, 2023, and December 26, 2025, indicating significant legal risks that may undermine shareholder confidence.
- Compensation Structure: Investors who bought shares during the class period may be entitled to compensation without any upfront costs, which could encourage more affected investors to join the lawsuit, potentially increasing the financial burden on the company.
- Counsel Selection Importance: The Rosen Law Firm emphasizes the necessity of choosing qualified legal representation, highlighting its past success in securing the largest securities class action settlement in 2017, thereby enhancing its credibility among investors.
- Case Details Unveiled: The lawsuit alleges that Ultragenyx made false or misleading statements regarding the potential of its drug setrusumab and the risks associated with study protocols, and if proven true, this could further damage the company's reputation and stock price.
See More
Class Action Lawsuit Announced for Ultragenyx Pharmaceutical
- Class Action Initiation: Rosen Law Firm has announced a class action lawsuit on behalf of investors who purchased Ultragenyx Pharmaceutical (NASDAQ: RARE) common stock between August 3, 2023, and December 26, 2025, highlighting the legal risks the company faces.
- Compensation Structure: Investors joining the class action will not incur any out-of-pocket fees, as the lawsuit operates on a contingency fee basis, thereby reducing financial barriers and encouraging more affected parties to seek justice.
- Lawsuit Allegations: The lawsuit claims that Ultragenyx concealed significant adverse facts regarding setrusumab, asserting that while it increases bone density, it does not correlate with reduced fracture rates, leading to investor losses when the truth emerged.
- Counsel Selection Advice: Investors are advised to choose qualified legal representation, with Rosen Law Firm recognized for its success in securities class actions, having recovered over $438 million for investors in 2019 alone, underscoring its credibility in the legal landscape.
See More
Class Action Lawsuit Filed Against Ultragenyx Pharmaceutical Inc.
- Lawsuit Background: Bronstein, Gewirtz & Grossman, LLC, a nationally recognized investor-rights law firm, has filed a class action lawsuit against Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and certain officers, seeking damages for investors who acquired Ultragenyx securities between August 3, 2023, and December 26, 2025.
- False Statements Allegation: The complaint alleges that throughout the class period, defendants made false and/or misleading statements and failed to disclose risks associated with the Phase III Orbit study for Osteogenesis Imperfecta (OI), leading investors to have an unwarranted optimistic view of the study's results.
- Investor Action: Affected investors are encouraged to apply to be lead plaintiffs by April 6, 2026, to share in any potential recovery, with the assurance that participation does not require serving as lead plaintiff.
- Law Firm's Reputation: Bronstein, Gewirtz & Grossman, LLC is well-regarded in securities fraud class actions, having recovered hundreds of millions for investors nationwide, emphasizing its commitment to restoring investor capital and ensuring corporate accountability.
See More
Ultragenyx Faces Class Action Lawsuit Over Stock Misrepresentation
- Class Action Initiation: Ultragenyx Pharmaceutical Inc. is facing a class action lawsuit for alleged violations of the Securities Exchange Act during stock transactions from August 3, 2023, to December 26, 2025, with investors required to apply as lead plaintiffs by April 6, 2025, indicating significant legal compliance risks for the company.
- Stock Price Volatility: Following the July 9, 2025, announcement that the Phase III Orbit study failed to achieve statistical significance, Ultragenyx's stock plummeted over 25%, and after the December 29, 2025, revelation of further study failures, the stock fell more than 42%, reflecting extreme market pessimism regarding the company's future.
- Study Result Failures: The lawsuit alleges that Ultragenyx failed to adequately communicate the risks associated with treatment efficacy in its Phase III Orbit study, severely undermining investor confidence in its products and potentially impacting future financing and market trust.
- Severe Legal Consequences: Robbins Geller LLP states that any investor who purchased Ultragenyx stock during the class period can seek lead plaintiff status, which, if successful, would represent all affected investors, highlighting the company's legal liabilities and potential compensation risks.
See More
Class Action Filed Against Ultragenyx Pharmaceutical Inc.
- Lawsuit Background: National plaintiffs' law firm Berger Montague PC has announced a class action lawsuit against Ultragenyx Pharmaceutical Inc. on behalf of investors who purchased stock between August 3, 2023, and December 26, 2025, indicating a significant loss of investor confidence in the company's future prospects.
- Stock Price Plunge: Following the disclosure on December 29, 2025, that Ultragenyx's ORBIT and COSMIC Phase 3 trials failed to meet their primary endpoints, the stock price plummeted over 42%, from $34.19 per share on December 26, 2025, to $19.72 per share, reflecting a pessimistic outlook from the market regarding the company's future.
- Investor Rights: Investors must apply by April 6, 2026, to be appointed as lead plaintiff representatives in the class action, highlighting concerns over corporate governance and information disclosure that may influence future investment decisions.
- Law Firm's Strength: Berger Montague, a leading law firm specializing in complex civil litigation and class actions, has recovered over $50 billion for clients over the past 55 years, showcasing its significant strength and influence in the legal field.
See More
Ultragenyx Resubmits BLA for Accelerated Approval of UX111 Gene Therapy
- Application Progress: On January 30, Ultragenyx Pharmaceutical resubmitted its Biologics License Application (BLA) to the FDA for accelerated approval of UX111 AAV9 gene therapy for Sanfilippo syndrome type A, with a review expected in Q3; if approved, it will be the first therapy for this condition.
- Market Analyst Support: Morgan Stanley analyst Maxwell Skor maintained a Buy rating on Ultragenyx with a price target of $50, reflecting confidence in the company's growth potential, while Leerink Partners also issued a Buy rating, indicating positive investor sentiment.
- Diverse Product Line: Ultragenyx focuses on therapies for rare and ultra-rare genetic diseases, with products like Crysvita, Mepsevii, Dojolvi, and Evkeeza, showcasing its broad market presence and potential in the biopharmaceutical sector.
- Investment Outlook Assessment: While RARE is viewed as a promising investment, analysts suggest that certain AI stocks may offer greater upside potential and lower downside risk, urging investors to carefully evaluate market dynamics to optimize their portfolios.
See More
Ultragenyx Pharmaceutical Class Action Lawsuit Announced
- Class Action Initiation: Rosen Law Firm has announced a class action lawsuit on behalf of investors who purchased Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) common stock between August 3, 2023, and December 26, 2025, indicating significant legal risks that may undermine shareholder confidence.
- Compensation Structure: Investors who bought shares during the class period may be entitled to compensation without any upfront costs, which could encourage more affected investors to join the lawsuit, potentially increasing the financial burden on the company.
- Counsel Selection Importance: The Rosen Law Firm emphasizes the necessity of choosing qualified legal representation, highlighting its past success in securing the largest securities class action settlement in 2017, thereby enhancing its credibility among investors.
- Case Details Unveiled: The lawsuit alleges that Ultragenyx made false or misleading statements regarding the potential of its drug setrusumab and the risks associated with study protocols, and if proven true, this could further damage the company's reputation and stock price.
See More
Class Action Lawsuit Announced for Ultragenyx Pharmaceutical
- Class Action Initiation: Rosen Law Firm has announced a class action lawsuit on behalf of investors who purchased Ultragenyx Pharmaceutical (NASDAQ: RARE) common stock between August 3, 2023, and December 26, 2025, highlighting the legal risks the company faces.
- Compensation Structure: Investors joining the class action will not incur any out-of-pocket fees, as the lawsuit operates on a contingency fee basis, thereby reducing financial barriers and encouraging more affected parties to seek justice.
- Lawsuit Allegations: The lawsuit claims that Ultragenyx concealed significant adverse facts regarding setrusumab, asserting that while it increases bone density, it does not correlate with reduced fracture rates, leading to investor losses when the truth emerged.
- Counsel Selection Advice: Investors are advised to choose qualified legal representation, with Rosen Law Firm recognized for its success in securities class actions, having recovered over $438 million for investors in 2019 alone, underscoring its credibility in the legal landscape.
See More
Class Action Lawsuit Filed Against Ultragenyx Pharmaceutical Inc.
- Lawsuit Background: Bronstein, Gewirtz & Grossman, LLC, a nationally recognized investor-rights law firm, has filed a class action lawsuit against Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and certain officers, seeking damages for investors who acquired Ultragenyx securities between August 3, 2023, and December 26, 2025.
- False Statements Allegation: The complaint alleges that throughout the class period, defendants made false and/or misleading statements and failed to disclose risks associated with the Phase III Orbit study for Osteogenesis Imperfecta (OI), leading investors to have an unwarranted optimistic view of the study's results.
- Investor Action: Affected investors are encouraged to apply to be lead plaintiffs by April 6, 2026, to share in any potential recovery, with the assurance that participation does not require serving as lead plaintiff.
- Law Firm's Reputation: Bronstein, Gewirtz & Grossman, LLC is well-regarded in securities fraud class actions, having recovered hundreds of millions for investors nationwide, emphasizing its commitment to restoring investor capital and ensuring corporate accountability.
See More
Ultragenyx Faces Class Action Lawsuit Over Stock Misrepresentation
- Class Action Initiation: Ultragenyx Pharmaceutical Inc. is facing a class action lawsuit for alleged violations of the Securities Exchange Act during stock transactions from August 3, 2023, to December 26, 2025, with investors required to apply as lead plaintiffs by April 6, 2025, indicating significant legal compliance risks for the company.
- Stock Price Volatility: Following the July 9, 2025, announcement that the Phase III Orbit study failed to achieve statistical significance, Ultragenyx's stock plummeted over 25%, and after the December 29, 2025, revelation of further study failures, the stock fell more than 42%, reflecting extreme market pessimism regarding the company's future.
- Study Result Failures: The lawsuit alleges that Ultragenyx failed to adequately communicate the risks associated with treatment efficacy in its Phase III Orbit study, severely undermining investor confidence in its products and potentially impacting future financing and market trust.
- Severe Legal Consequences: Robbins Geller LLP states that any investor who purchased Ultragenyx stock during the class period can seek lead plaintiff status, which, if successful, would represent all affected investors, highlighting the company's legal liabilities and potential compensation risks.
See More
Class Action Filed Against Ultragenyx Pharmaceutical Inc.
- Lawsuit Background: National plaintiffs' law firm Berger Montague PC has announced a class action lawsuit against Ultragenyx Pharmaceutical Inc. on behalf of investors who purchased stock between August 3, 2023, and December 26, 2025, indicating a significant loss of investor confidence in the company's future prospects.
- Stock Price Plunge: Following the disclosure on December 29, 2025, that Ultragenyx's ORBIT and COSMIC Phase 3 trials failed to meet their primary endpoints, the stock price plummeted over 42%, from $34.19 per share on December 26, 2025, to $19.72 per share, reflecting a pessimistic outlook from the market regarding the company's future.
- Investor Rights: Investors must apply by April 6, 2026, to be appointed as lead plaintiff representatives in the class action, highlighting concerns over corporate governance and information disclosure that may influence future investment decisions.
- Law Firm's Strength: Berger Montague, a leading law firm specializing in complex civil litigation and class actions, has recovered over $50 billion for clients over the past 55 years, showcasing its significant strength and influence in the legal field.
See More
