UX111 Poised to Be First Therapy for Sanfilippo Syndrome Type A
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Apr 02 2026
0mins
Should l Buy RARE?
Source: Newsfilter
- FDA Review Progress: Ultragenyx's Biologics License Application (BLA) for UX111 has been accepted for review by the FDA, with a decision expected by September 19, 2026, marking a significant step towards providing the first effective treatment for Sanfilippo syndrome Type A, which could greatly benefit affected families.
- Clinical Data Support: Updated long-term clinical data indicate that UX111 demonstrates durable treatment effects across multiple biomarker evaluations while maintaining an acceptable safety profile, providing robust support for the FDA's accelerated approval process, which could significantly enhance patient quality of life.
- Manufacturing Capability Assurance: If approved, UX111 will be manufactured at Andelyn Biosciences and Ultragenyx's facilities in the U.S., ensuring product quality and supply chain stability, while also accelerating market entry to meet urgent patient needs.
- Significant Market Potential: Sanfilippo syndrome Type A affects approximately 3,000 to 5,000 patients with no current effective treatments; the successful launch of UX111 would fill this market gap, potentially yielding substantial economic returns and market share for Ultragenyx.
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Analyst Views on RARE
Wall Street analysts forecast RARE stock price to rise
18 Analyst Rating
17 Buy
1 Hold
0 Sell
Strong Buy
Current: 25.060
Low
35.00
Averages
61.65
High
120.00
Current: 25.060
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.
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- Emotional and Professional Intertwining: As a physician, facing his son's rare disease brought profound grief and a sense of loss of control, which transformed his emotional landscape and deepened his empathy and understanding for patients' suffering in his medical career.
- Patience and Growth: Gabe's condition taught Dr. D'Orazio invaluable lessons in patience and a reevaluation of life, enhancing his focus on emotional needs in medical practice and significantly improving his empathy as a physician.
- Community and Advocacy: By engaging with the Foundation for Angelman Syndrome Therapeutics, Dr. D'Orazio transformed feelings of isolation into connection, aiding other families and advancing research, showcasing the resilience and unity of families affected by rare diseases.
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- False Statements Allegations: The complaint alleges that Ultragenyx made overly optimistic statements regarding the expected results of its Phase III Orbit and Cosmic studies for setrusumab, despite actual data showing the drug failed to significantly reduce fracture rates, leading investors to purchase shares at inflated prices and incur substantial losses.
- Stock Price Plummet: Following the announcement on December 29, 2025, that the studies did not achieve primary endpoints, Ultragenyx's stock price fell from $34.19 to $19.72, a staggering 42.32% drop in a single day, reflecting a severe loss of market confidence in the company's prospects.
- Investor Rights Protection: Investors who suffered losses during the class period can apply to be lead plaintiffs in the lawsuit by April 6, 2026, demonstrating that legal avenues are available for affected investors seeking potential remedies.
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- False Statements Exposed: The complaint alleges that Ultragenyx made false and misleading statements regarding its understanding of the effects of its drug candidate on patients with Osteogenesis Imperfecta, leading to an overly optimistic outlook among investors.
- Study Results Impact: The Phase III ORBIT study revealed that Ultragenyx failed to achieve a statistically significant reduction in annualized fracture rate, highlighting significant errors in the company's drug efficacy claims and resulting in investor losses.
- Legal Consultation Opportunity: Investors are encouraged to contact the Schall Law Firm before April 6, 2026, to discuss their rights and participate in the lawsuit to seek compensation for their losses.
See More
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- Class Action Deadline: Investors who purchased Ultragenyx common stock between August 3, 2023, and December 26, 2025, must submit their applications by April 6, 2026, to participate in the class action, as failing to do so may result in losing their right to compensation.
- Lawsuit Background: The lawsuit alleges that the company provided overly optimistic expectations regarding setrusumab's efficacy in Phase III Orbit and Cosmic studies, leading investors to purchase shares at inflated prices, which ultimately caused financial losses when the true results were revealed, highlighting the importance of accurate information disclosure.
- Law Firm Credentials: Rosen Law Firm specializes in securities class actions and has recovered over $438 million for investors in 2019 alone, demonstrating its successful track record in similar cases, thus investors should carefully select their legal counsel.
- Investor Action Steps: Investors can visit the Rosen Law Firm's website or call the toll-free number for more information on joining the class action, ensuring they receive appropriate legal representation as the case progresses.
See More
Class Action Lawsuit Filed Against Ultragenyx Pharmaceutical Inc.
- Lawsuit Background: Bronstein, Gewirtz & Grossman, LLC has filed a class action lawsuit against Ultragenyx Pharmaceutical Inc., alleging violations of federal securities laws affecting all investors who purchased securities between August 3, 2023, and December 26, 2025.
- False Statement Allegations: The complaint claims that throughout the class period, defendants made false and/or misleading statements and failed to disclose risks associated with their Phase III Orbit study, leading investors to have an inaccurate understanding of the drug setrusumab's efficacy.
- 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 they do not need to serve as lead plaintiffs to participate in any compensation.
- Fee Arrangement: Bronstein, Gewirtz & Grossman, LLC represents investors on a contingency fee basis, meaning they will only seek reimbursement for expenses and attorney fees if they are successful, minimizing the financial burden on investors.
See More
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- Lawsuit Background: The lawsuit alleges that defendants provided false and misleading information regarding setrusumab (UX 143) during Phase III Orbit and Cosmic studies, leading investors to purchase shares at artificially inflated prices.
- Choosing Legal Representation: Rosen Law Firm emphasizes the importance of selecting qualified counsel with a successful track record, noting that many firms issuing notices lack the necessary resources and experience to effectively litigate securities class actions.
- Investor Losses: The lawsuit claims that Ultragenyx shareholders suffered losses due to the defendants' false statements, particularly after the true information entered the market, resulting in a significant decline in stock prices and substantial financial losses for investors.
See More
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- Family Life Restructured: Dr. Joseph D'Orazio's family life was immediately altered upon learning of his son Gabe's diagnosis of Angelman syndrome, necessitating a complete reevaluation of daily routines and future plans to accommodate the realities of a special needs family.
- Emotional and Professional Intertwining: As a physician, facing his son's rare disease brought profound grief and a sense of loss of control, which transformed his emotional landscape and deepened his empathy and understanding for patients' suffering in his medical career.
- Patience and Growth: Gabe's condition taught Dr. D'Orazio invaluable lessons in patience and a reevaluation of life, enhancing his focus on emotional needs in medical practice and significantly improving his empathy as a physician.
- Community and Advocacy: By engaging with the Foundation for Angelman Syndrome Therapeutics, Dr. D'Orazio transformed feelings of isolation into connection, aiding other families and advancing research, showcasing the resilience and unity of families affected by rare diseases.
See More
Ultragenyx Faces Class Action Lawsuit Over Stock Losses
- Class Action Initiated: Bragar Eagel & Squire, P.C. has filed a class action lawsuit against Ultragenyx in the Northern District of California on behalf of investors who purchased shares between August 3, 2023, and December 26, 2025, indicating significant investor dissatisfaction with the company's future outlook.
- False Statements Allegations: The complaint alleges that Ultragenyx made overly optimistic statements regarding the expected results of its Phase III Orbit and Cosmic studies for setrusumab, despite actual data showing the drug failed to significantly reduce fracture rates, leading investors to purchase shares at inflated prices and incur substantial losses.
- Stock Price Plummet: Following the announcement on December 29, 2025, that the studies did not achieve primary endpoints, Ultragenyx's stock price fell from $34.19 to $19.72, a staggering 42.32% drop in a single day, reflecting a severe loss of market confidence in the company's prospects.
- Investor Rights Protection: Investors who suffered losses during the class period can apply to be lead plaintiffs in the lawsuit by April 6, 2026, demonstrating that legal avenues are available for affected investors seeking potential remedies.
See More
Class Action Lawsuit Reminder Against Ultragenyx Pharmaceutical Inc.
- Class Action Notice: The Schall Law Firm reminds investors of a class action lawsuit against Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) for violations of securities laws during the period from August 3, 2023, to December 26, 2025.
- False Statements Exposed: The complaint alleges that Ultragenyx made false and misleading statements regarding its understanding of the effects of its drug candidate on patients with Osteogenesis Imperfecta, leading to an overly optimistic outlook among investors.
- Study Results Impact: The Phase III ORBIT study revealed that Ultragenyx failed to achieve a statistically significant reduction in annualized fracture rate, highlighting significant errors in the company's drug efficacy claims and resulting in investor losses.
- Legal Consultation Opportunity: Investors are encouraged to contact the Schall Law Firm before April 6, 2026, to discuss their rights and participate in the lawsuit to seek compensation for their losses.
See More
Ultragenyx Pharmaceutical Class Action Notice
- Class Action Deadline: Investors who purchased Ultragenyx common stock between August 3, 2023, and December 26, 2025, must submit their applications by April 6, 2026, to participate in the class action, as failing to do so may result in losing their right to compensation.
- Lawsuit Background: The lawsuit alleges that the company provided overly optimistic expectations regarding setrusumab's efficacy in Phase III Orbit and Cosmic studies, leading investors to purchase shares at inflated prices, which ultimately caused financial losses when the true results were revealed, highlighting the importance of accurate information disclosure.
- Law Firm Credentials: Rosen Law Firm specializes in securities class actions and has recovered over $438 million for investors in 2019 alone, demonstrating its successful track record in similar cases, thus investors should carefully select their legal counsel.
- Investor Action Steps: Investors can visit the Rosen Law Firm's website or call the toll-free number for more information on joining the class action, ensuring they receive appropriate legal representation as the case progresses.
See More
Class Action Lawsuit Filed Against Ultragenyx Pharmaceutical Inc.
- Lawsuit Background: Bronstein, Gewirtz & Grossman, LLC has filed a class action lawsuit against Ultragenyx Pharmaceutical Inc., alleging violations of federal securities laws affecting all investors who purchased securities between August 3, 2023, and December 26, 2025.
- False Statement Allegations: The complaint claims that throughout the class period, defendants made false and/or misleading statements and failed to disclose risks associated with their Phase III Orbit study, leading investors to have an inaccurate understanding of the drug setrusumab's efficacy.
- 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 they do not need to serve as lead plaintiffs to participate in any compensation.
- Fee Arrangement: Bronstein, Gewirtz & Grossman, LLC represents investors on a contingency fee basis, meaning they will only seek reimbursement for expenses and attorney fees if they are successful, minimizing the financial burden on investors.
See More
Ultragenyx Pharmaceutical Class Action Reminder
- Class Action Notice: Rosen Law Firm reminds investors who purchased Ultragenyx (NASDAQ:RARE) common stock between August 3, 2023, and December 26, 2025, to apply as lead plaintiffs by April 6, 2026, to participate in the class action and seek compensation.
- Lawsuit Background: The lawsuit alleges that defendants provided false and misleading information regarding setrusumab (UX 143) during Phase III Orbit and Cosmic studies, leading investors to purchase shares at artificially inflated prices.
- Choosing Legal Representation: Rosen Law Firm emphasizes the importance of selecting qualified counsel with a successful track record, noting that many firms issuing notices lack the necessary resources and experience to effectively litigate securities class actions.
- Investor Losses: The lawsuit claims that Ultragenyx shareholders suffered losses due to the defendants' false statements, particularly after the true information entered the market, resulting in a significant decline in stock prices and substantial financial losses for investors.
See More
