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Ultragenyx Pharmaceutical Stock (NASDAQ: RARE)

Dernières nouvelles pour Ultragenyx Pharmaceutical (NASDAQ:RARE) Stock

ULTRAGENYX PHARMACEUTICAL IN

Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical Inc is a USA-based biopharmaceutical company. It identifies, acquires, develops, and commercializes novel products for the treatment of serious rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. The company's medicine portfolio includes Crysvita, Dojolvi and Mepsevii. Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 year of age and older. Mepsevii is indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII.

News

RARE

Ultragenyx Receives FDA Breakthrough Therapy Designation For GTX-102 In Angelman Syndrome

4 analysts have expressed a variety of opinions on Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) over the past quarter, offering a diverse set of opinions from bullish to bearish.
The following table summarizes their recent ratings, shedding light on the changing sentiments within the past 30 days and comparing them to the preceding months.
<table>
<thead>
<tr>
<th></th>
<th align="right">Bullish</th>
<th align="right">Somewhat Bullish</th>
<th align="right">Indifferent</th>
<th align="right">Somewhat Bearish</th>
<th align="right">Bearish</th>
</tr>
</thead>
<tbody>
<tr>
<td>Total Ratings</td>
<td align="right">1</td>
<td align="right">3</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>Last 30D</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>1M Ago</td>
<td align="right">0</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>2M Ago</td>
<td align="right">0</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>3M Ago</td>
<td align="right">0</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
</tbody>
</table>
The 12-month price targets assessed by analysts reveal further insights, featuring an average target of $77.75, a high estimate of $117.00, and a low estimate of $64.00. This current average reflects an increase of 0.54% from the previous average price target of $77.33. 
<img src="https://www.benzinga.com/files/images/story/2025/1750428056_0.png" alt="price target chart" />
<h2>Diving into Analyst Ratings: An In-Depth Exploration</h2>
The analysis of recent analyst actions sheds light on the perception of Ultragenyx Pharmaceutical by financial experts. The following summary presents key analysts, their recent evaluations, and adjustments to ratings and price targets.
<table>
<thead>
<tr>
<th>Analyst</th>
<th>Analyst Firm</th>
<th>Action Taken</th>
<th>Rating</th>
<th>Current Price Target</th>
<th>Prior Price Target</th>
</tr>
</thead>
<tbody>
<tr>
<td>Debjit Chattopadhyay</td>
<td>Guggenheim</td>
<td>Maintains</td>
<td>Buy</td>
<td>$64.00</td>
<td>$64.00</td>
</tr>
<tr>
<td>Sami Corwin</td>
<td>William Blair</td>
<td>Announces</td>
<td>Outperform</td>
<td>$65.00</td>
<td>-</td>
</tr>
<tr>
<td>Jeffrey Hung</td>
<td>Morgan Stanley</td>
<td>Raises</td>
<td>Overweight</td>
<td>$65.00</td>
<td>$64.00</td>
</tr>
<tr>
<td>Anupam Rama</td>
<td>JP Morgan</td>
<td>Raises</td>
<td>Overweight</td>
<td>$117.00</td>
<td>$104.00</td>
</tr>
</tbody>
</table>
<h3>Key Insights:</h3>
<ul>
<li>Action Taken: In response to dynamic market conditions and company performance, analysts update their recommendations. Whether they 'Maintain', 'Raise', or 'Lower' their stance, it signifies their reaction to recent developments related to Ultragenyx Pharmaceutical. This insight gives a snapshot of analysts' perspectives on the current state of the company.</li>
<li>Rating: Analysts assign qualitative assessments to stocks, ranging from 'Outperform' to 'Underperform'. These ratings convey the analysts' expectations for the relative performance of Ultragenyx Pharmaceutical compared to the broader market.</li>
<li>Price Targets: Analysts gauge the dynamics of price targets, providing estimates for the future value of Ultragenyx Pharmaceutical's stock. This comparison reveals trends in analysts' expectations over time.</li>
</ul>
Capture valuable insights into Ultragenyx Pharmaceutical's market standing by understanding these analyst evaluations alongside pertinent financial indicators. Stay informed and make strategic decisions with our Ratings Table.
<a href="https://www.benzinga.com/quote/RARE/analyst-ratings?adType=benzinga-insights&amp;ad=analyst-ratings&amp;campaign=wallstreetadvantage">Stay up to date on Ultragenyx Pharmaceutical analyst ratings.</a>
<h2>All You Need to Know About Ultragenyx Pharmaceutical</h2>
Ultragenyx Pharmaceutical Inc is a USA-based biopharmaceutical company. It identifies, acquires, develops, and commercializes novel products for the treatment of serious rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. The company's medicine portfolio includes Crysvita, Dojolvi and Mepsevii. Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 year of age and older. Mepsevii is indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII.
<h3>Breaking Down Ultragenyx Pharmaceutical's Financial Performance</h3>
Market Capitalization Analysis: Falling below industry benchmarks, the company's market capitalization reflects a reduced size compared to peers. This positioning may be influenced by factors such as growth expectations or operational capacity.
Revenue Growth: Ultragenyx Pharmaceutical's remarkable performance in 3M is evident. As of 31 March, 2025, the company achieved an impressive revenue growth rate of 27.99%. This signifies a substantial increase in the company's top-line earnings. As compared to its peers, the revenue growth lags behind its industry peers. The company achieved a growth rate lower than the average among peers in Health Care sector.
Net Margin: Ultragenyx Pharmaceutical's net margin is impressive, surpassing industry averages. With a net margin of -108.46%, the company demonstrates strong profitability and effective cost management.
Return on Equity (ROE): Ultragenyx Pharmaceutical's ROE is below industry standards, pointing towards difficulties in efficiently utilizing equity capital. With an ROE of -75.63%, the company may encounter challenges in delivering satisfactory returns for shareholders.
Return on Assets (ROA): Ultragenyx Pharmaceutical's ROA lags behind industry averages, suggesting challenges in maximizing returns from its assets. With an ROA of -10.73%, the company may face hurdles in achieving optimal financial performance.
Debt Management: Ultragenyx Pharmaceutical's debt-to-equity ratio is notably higher than the industry average. With a ratio of 0.27, the company relies more heavily on borrowed funds, indicating a higher level of financial risk.
<h2>The Significance of Analyst Ratings Explained</h2>
Experts in banking and financial systems, analysts specialize in reporting for specific stocks or defined sectors. Their comprehensive research involves attending company conference calls and meetings, analyzing financial statements, and engaging with insiders to generate what are known as analyst ratings for stocks. Typically, analysts assess and rate each stock once per quarter.
Analysts may supplement their ratings with predictions for metrics like growth estimates, earnings, and revenue, offering investors a more comprehensive outlook. However, investors should be mindful that analysts, like any human, can have subjective perspectives influencing their forecasts.
Which Stocks Are Analysts Recommending Now?
Benzinga Edge gives you instant access to all major analyst upgrades, downgrades, and price targets. Sort by accuracy, upside potential, and more. <a href="https://www.benzinga.com/premium/ideas/benzinga-edge-checkout/?t=be8be9arja3be22bz21&amp;adType=benzinga-insights&amp;ad=analyst-ratings&amp;campaign=wallstreetadvantage">Click here to stay ahead of the market</a>.
This article was generated by Benzinga's automated content engine and reviewed by an editor.

Analyst Ratings

Ultragenyx Pharmaceutical Stock: A Deep Dive Into Analyst Perspectives (4 Ratings)

Guggenheim analyst Debjit Chattopadhyay reiterates Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) with a Buy and maintains $64 price target.

Price Target

Reiteration

Guggenheim Reiterates Buy on Ultragenyx Pharmaceutical, Maintains $64 Price Target

Ratings for Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) were provided by 4 analysts in the past three months, showcasing a mix of bullish and bearish perspectives.
Summarizing their recent assessments, the table below illustrates the evolving sentiments in the past 30 days and compares them to the preceding months.
<table>
<thead>
<tr>
<th></th>
<th align="right">Bullish</th>
<th align="right">Somewhat Bullish</th>
<th align="right">Indifferent</th>
<th align="right">Somewhat Bearish</th>
<th align="right">Bearish</th>
</tr>
</thead>
<tbody>
<tr>
<td>Total Ratings</td>
<td align="right">0</td>
<td align="right">4</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>Last 30D</td>
<td align="right">0</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>1M Ago</td>
<td align="right">0</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>2M Ago</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>3M Ago</td>
<td align="right">0</td>
<td align="right">2</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
</tbody>
</table>
Insights from analysts' 12-month price targets are revealed, presenting an average target of $90.5, a high estimate of $117.00, and a low estimate of $65.00. This current average has decreased by 11.85% from the previous average price target of $102.67. 
<img src="https://www.benzinga.com/files/images/story/2025/1748462428_0.png" alt="price target chart" />
<h2>Breaking Down Analyst Ratings: A Detailed Examination</h2>
In examining recent analyst actions, we gain insights into how financial experts perceive Ultragenyx Pharmaceutical. The following summary outlines key analysts, their recent evaluations, and adjustments to ratings and price targets.
<table>
<thead>
<tr>
<th>Analyst</th>
<th>Analyst Firm</th>
<th>Action Taken</th>
<th>Rating</th>
<th>Current Price Target</th>
<th>Prior Price Target</th>
</tr>
</thead>
<tbody>
<tr>
<td>Sami Corwin</td>
<td>William Blair</td>
<td>Announces</td>
<td>Outperform</td>
<td>$65.00</td>
<td>-</td>
</tr>
<tr>
<td>Jeffrey Hung</td>
<td>Morgan Stanley</td>
<td>Raises</td>
<td>Overweight</td>
<td>$65.00</td>
<td>$64.00</td>
</tr>
<tr>
<td>Anupam Rama</td>
<td>JP Morgan</td>
<td>Raises</td>
<td>Overweight</td>
<td>$117.00</td>
<td>$104.00</td>
</tr>
<tr>
<td>Allison Bratzel</td>
<td>Piper Sandler</td>
<td>Lowers</td>
<td>Overweight</td>
<td>$115.00</td>
<td>$140.00</td>
</tr>
</tbody>
</table>
<h3>Key Insights:</h3>
<ul>
<li>Action Taken: Analysts adapt their recommendations to changing market conditions and company performance. Whether they 'Maintain', 'Raise' or 'Lower' their stance, it reflects their response to recent developments related to Ultragenyx Pharmaceutical. This information provides a snapshot of how analysts perceive the current state of the company.</li>
<li>Rating: Analysts unravel qualitative evaluations for stocks, ranging from 'Outperform' to 'Underperform'. These ratings offer insights into expectations for the relative performance of Ultragenyx Pharmaceutical compared to the broader market.</li>
<li>Price Targets: Analysts gauge the dynamics of price targets, providing estimates for the future value of Ultragenyx Pharmaceutical's stock. This comparison reveals trends in analysts' expectations over time.</li>
</ul>
Navigating through these analyst evaluations alongside other financial indicators can contribute to a holistic understanding of Ultragenyx Pharmaceutical's market standing. Stay informed and make data-driven decisions with our Ratings Table.
<a href="https://www.benzinga.com/quote/RARE/analyst-ratings?adType=benzinga-insights&amp;ad=analyst-ratings&amp;campaign=wallstreetadvantage">Stay up to date on Ultragenyx Pharmaceutical analyst ratings.</a>
<h2>Unveiling the Story Behind Ultragenyx Pharmaceutical</h2>
Ultragenyx Pharmaceutical Inc is a USA-based biopharmaceutical company. It identifies, acquires, develops, and commercializes novel products for the treatment of serious rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. The company's medicine portfolio includes Crysvita, Dojolvi and Mepsevii. Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 year of age and older. Mepsevii is indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII.
<h3>Breaking Down Ultragenyx Pharmaceutical's Financial Performance</h3>
Market Capitalization Analysis: The company's market capitalization surpasses industry averages, showcasing a dominant size relative to peers and suggesting a strong market position.
Revenue Growth: Ultragenyx Pharmaceutical displayed positive results in 3M. As of 31 March, 2025, the company achieved a solid revenue growth rate of approximately 27.99%. This indicates a notable increase in the company's top-line earnings. When compared to others in the Health Care sector, the company excelled with a growth rate higher than the average among peers.
Net Margin: The company's net margin is a standout performer, exceeding industry averages. With an impressive net margin of -108.46%, the company showcases strong profitability and effective cost control.
Return on Equity (ROE): Ultragenyx Pharmaceutical's ROE falls below industry averages, indicating challenges in efficiently using equity capital. With an ROE of -75.63%, the company may face hurdles in generating optimal returns for shareholders.
Return on Assets (ROA): Ultragenyx Pharmaceutical's ROA lags behind industry averages, suggesting challenges in maximizing returns from its assets. With an ROA of -10.73%, the company may face hurdles in achieving optimal financial performance.
Debt Management: The company faces challenges in debt management with a debt-to-equity ratio higher than the industry average. With a ratio of 0.27, caution is advised due to increased financial risk.
<h2>The Significance of Analyst Ratings Explained</h2>
Experts in banking and financial systems, analysts specialize in reporting for specific stocks or defined sectors. Their comprehensive research involves attending company conference calls and meetings, analyzing financial statements, and engaging with insiders to generate what are known as analyst ratings for stocks. Typically, analysts assess and rate each stock once per quarter.
Some analysts publish their predictions for metrics such as growth estimates, earnings, and revenue to provide additional guidance with their ratings. When using analyst ratings, it is important to keep in mind that stock and sector analysts are also human and are only offering their opinions to investors.
If you want to keep track of which analysts are outperforming others, you can view updated analyst ratings along withanalyst success scores in <a href="https://www.benzinga.com/pro/">Benzinga Pro</a>.
Which Stocks Are Analysts Recommending Now?
Benzinga Edge gives you instant access to all major analyst upgrades, downgrades, and price targets. Sort by accuracy, upside potential, and more. <a href="https://www.benzinga.com/premium/ideas/benzinga-edge-checkout/?t=be8be9arja3be22bz21&amp;adType=benzinga-insights&amp;ad=analyst-ratings&amp;campaign=wallstreetadvantage">Click here to stay ahead of the market</a>.
This article was generated by Benzinga's automated content engine and reviewed by an editor.

What 4 Analyst Ratings Have To Say About Ultragenyx Pharmaceutical

On Wednesday, William Blair initiated coverage on Ultragenyx Pharmaceutical Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>), a biopharmaceutical company focused on developing and commercializing therapies for rare and ultra-<a href="https://www.benzinga.com/general/health-care" target="_blank" rel="noreferrer noopener">rare genetic diseases</a>.
Since 2017, the company has commercialized four products, which generated $560 million in revenue in 2024, up 29% year-over-year. In the first quarter of 2025, sales increased <a href="https://www.benzinga.com/pressreleases/25/05/g45234765/ultragenyx-reports-first-quarter-2025-financial-results-and-corporate-update" target="_blank" rel="noreferrer noopener">28% to $139.29 million</a>.
Ultragenyx expects fiscal 2025 sales of $640 million-$670 million versus <a target="_blank" href="https://www.benzinga.com/calendars/earnings" rel="noreferrer noopener">consensus of $655.02 million</a>.
&#8220;We anticipate Ultragenyx&#8217;s legacy products will continue to provide a strong revenue foundation for the company through the early 2030s,&#8221; writes William Blair analyst Sami Corwin.
Also Read: <a target="_blank" href="https://www.benzinga.com/general/biotech/25/03/44523017/ultragenyx-is-uniquely-positioned-in-long-term-remains-on-jpmorgans-focus-list" rel="noreferrer noopener">Ultragenyx Stays On JPMorgan&#8217;s Focus List As Analyst Highlights Strong Long-Term Positioning</a>
William Blair initiated coverage <a href="https://www.benzinga.com/quote/RARE/analyst-ratings">with an Outperform rating</a>, writing that the company is at an inflection point. Potentially, six additional therapeutics will be approved by the end of 2028, including three approvals in the next 18 months.
Corwin notes that these additional assets synergize with Ultragenyx&#8217;s existing commercial portfolio, aiding in strong commercial launches. She projects the company&#8217;s expanding revenue stream, supplemented by the potential receipt of three priority review vouchers, could enable the company to achieve GAAP profitability in 2027 and beyond.
William Blair noted that regulatory decisions and clinical data could be catalysts for the stock in the near term. Specifically, positive results from the Phase 3 trial of setrusumab in osteogenesis imperfecta (OI) could lead to significant upside.
Corwin projects the products could begin contributing to the top line as early as the fourth quarter of 2025 and could lead to a major revenue inflection in 2026 with the potential approval of pertuzumab in OI.
William Blair views setrusumab as a blockbuster product and estimates peak revenue of $2.4 billion in 2037, $1.4 billion of which will go to Ultragenyx.
Price Action: RARE stock is down 1.65% at $34.50 at the last check Wednesday.
Read Next:
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</ul>
Photo: Shutterstock

Analyst Color

Biotech

Health Care

Initiation

General

Analyst Bullish On Ultragenyx, Poised For Growth With Six Drug Approvals Expected By 2028

William Blair analyst Sami Corwin initiates coverage on Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) with a Outperform rating and announces Price Target of $65.

William Blair Initiates Coverage On Ultragenyx Pharmaceutical with Outperform Rating, Announces Price Target of $65

Morgan Stanley analyst Jeffrey Hung maintains Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) with a Overweight and raises the price target from $64 to $65.

Morgan Stanley Maintains Overweight on Ultragenyx Pharmaceutical, Raises Price Target to $65

WIIM

RARE stock has given up its prior gain. Ultragenyx Pharmaceutical shares were trading higher after the company reported better-than-expected Q1 EPS results.

Ultragenyx Pharmaceutical shares are trading higher after the company reported better-than-expected Q1 EPS results.

Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) affirms FY2025 sales outlook from $640.00 million-$670.00 million to $640.00 million-$670.00 million vs $655.02 million estimate.

Guidance

Ultragenyx Pharmaceutical Affirms FY2025 Sales Guidance of $640.00M-$670.00M vs $655.02M Est

Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) reported quarterly losses of $(1.57) per share which beat the analyst consensus estimate of $(1.59) by 1.26 percent. This is a 22.66 percent increase over losses of $(2.03) per share from the same period last year. The company reported quarterly sales of $139.29 million which missed the analyst consensus estimate of $143.83 million by 3.15 percent. This is a 27.99 percent increase over sales of $108.83 million the same period last year.

Earnings

Earnings Beats

Earnings Misses

Ultragenyx Pharmaceutical Q1 EPS $(1.57) Beats $(1.59) Estimate, Sales $139.29M Miss $143.83M Estimate

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Earnings Scheduled For May 6, 2025

Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) is set to give its latest quarterly earnings report on Tuesday, 2025-05-06. Here's what investors need to know before the announcement.
Analysts estimate that Ultragenyx Pharmaceutical will report an earnings per share (EPS) of $-1.62. 
Investors in Ultragenyx Pharmaceutical are eagerly awaiting the company's announcement, hoping for news of surpassing estimates and positive guidance for the next quarter.
It's worth noting for new investors that stock prices can be heavily influenced by future projections rather than just past performance.
<h3>Past Earnings Performance</h3>
The company's EPS missed by $0.12 in the last quarter, leading to a 2.37% drop in the share price on the following day.
Here's a look at Ultragenyx Pharmaceutical's past performance and the resulting price change:

<table>
<thead>
<tr>
<th>Quarter</th>
<th>Q4 2024</th>
<th>Q3 2024</th>
<th>Q2 2024</th>
<th>Q1 2024</th>
</tr>
</thead>
<tbody>
<tr>
<td>EPS Estimate</td>
<td>-1.27</td>
<td>-1.45</td>
<td>-1.72</td>
<td>-1.76</td>
</tr>
<tr>
<td>EPS Actual</td>
<td>-1.39</td>
<td>-1.40</td>
<td>-1.52</td>
<td>-2.03</td>
</tr>
<tr>
<td>Price Change %</td>
<td>-2.0%</td>
<td>-3.0%</td>
<td>14.000000000000002%</td>
<td>-1.0%</td>
</tr>
</tbody>
</table>

<img src="https://www.benzinga.com/files/images/story/2025/1746464483_0.png" alt="eps graph" />
<h3>Performance of Ultragenyx Pharmaceutical Shares</h3>
Shares of Ultragenyx Pharmaceutical were trading at $39.3 as of May 02. Over the last 52-week period, shares are down 6.76%. Given that these returns are generally negative, long-term shareholders are likely upset going into this earnings release. 
<h3>Analyst Insights on Ultragenyx Pharmaceutical</h3>
For investors, grasping market sentiments and expectations in the industry is vital. This analysis explores the latest insights regarding Ultragenyx Pharmaceutical.
Analysts have provided Ultragenyx Pharmaceutical with 6 ratings, resulting in a consensus rating of Buy. The average one-year price target stands at $112.67, suggesting a potential 186.69% upside.
<h3>Comparing Ratings with Peers</h3>
The below comparison of the analyst ratings and average 1-year price targets of SpringWorks Therapeutics, Rhythm Pharmaceuticals and Catalyst Pharmaceuticals, three prominent players in the industry, gives insights for their relative performance expectations and market positioning.
<ul>
<li>Analysts currently favor an Neutral trajectory for SpringWorks Therapeutics, with an average 1-year price target of $61.33, suggesting a potential 56.06% upside.</li>
<li>Analysts currently favor an Buy trajectory for Rhythm Pharmaceuticals, with an average 1-year price target of $73.29, suggesting a potential 86.49% upside.</li>
<li>Analysts currently favor an Outperform trajectory for Catalyst Pharmaceuticals, with an average 1-year price target of $32.0, suggesting a potential 18.58% downside.</li>
</ul>
<h3>Peer Metrics Summary</h3>
The peer analysis summary provides a snapshot of key metrics for SpringWorks Therapeutics, Rhythm Pharmaceuticals and Catalyst Pharmaceuticals, illuminating their respective standings within the industry. These metrics offer valuable insights into their market positions and comparative performance.

<table>
<thead>
<tr>
<th>Company</th>
<th>Consensus</th>
<th>Revenue Growth</th>
<th>Gross Profit</th>
<th>Return on Equity</th>
</tr>
</thead>
<tbody>
<tr>
<td>Ultragenyx Pharmaceutical</td>
<td>Buy</td>
<td>29.42%</td>
<td>$147.98M</td>
<td>-44.30%</td>
</tr>
<tr>
<td>SpringWorks Therapeutics</td>
<td>Neutral</td>
<td>1029.96%</td>
<td>$55.99M</td>
<td>-15.25%</td>
</tr>
<tr>
<td>Rhythm Pharmaceuticals</td>
<td>Buy</td>
<td>72.61%</td>
<td>$38.04M</td>
<td>-286.92%</td>
</tr>
<tr>
<td>Catalyst Pharmaceuticals</td>
<td>Outperform</td>
<td>28.27%</td>
<td>$120.18M</td>
<td>8.06%</td>
</tr>
</tbody>
</table>

Key Takeaway:
Ultragenyx Pharmaceutical ranks at the bottom for Revenue Growth and Gross Profit, while it ranks at the top for Return on Equity. Overall, it is positioned in the middle compared to its peers.
<h3>Get to Know Ultragenyx Pharmaceutical Better</h3>
Ultragenyx Pharmaceutical Inc is a USA-based biopharmaceutical company. It identifies, acquires, develops, and commercializes novel products for the treatment of serious rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. The company's medicine portfolio includes Crysvita, Dojolvi and Mepsevii. Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 year of age and older. Mepsevii is indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII.
<h3>Breaking Down Ultragenyx Pharmaceutical's Financial Performance</h3>
Market Capitalization Analysis: Below industry benchmarks, the company's market capitalization reflects a smaller scale relative to peers. This could be attributed to factors such as growth expectations or operational capacity.
Revenue Growth: Ultragenyx Pharmaceutical displayed positive results in 3 months. As of 31 December, 2024, the company achieved a solid revenue growth rate of approximately 29.42%. This indicates a notable increase in the company's top-line earnings. When compared to others in the Health Care sector, the company faces challenges, achieving a growth rate lower than the average among peers.
Net Margin: Ultragenyx Pharmaceutical's net margin is impressive, surpassing industry averages. With a net margin of -80.9%, the company demonstrates strong profitability and effective cost management.
Return on Equity (ROE): Ultragenyx Pharmaceutical's ROE falls below industry averages, indicating challenges in efficiently using equity capital. With an ROE of -44.3%, the company may face hurdles in generating optimal returns for shareholders.
Return on Assets (ROA): The company's ROA is below industry benchmarks, signaling potential difficulties in efficiently utilizing assets. With an ROA of -8.77%, the company may need to address challenges in generating satisfactory returns from its assets.
Debt Management: Ultragenyx Pharmaceutical's debt-to-equity ratio is below the industry average at 0.16, reflecting a lower dependency on debt financing and a more conservative financial approach.
<a href="https://www.benzinga.com/quote/RARE/earnings">To track all earnings releases for Ultragenyx Pharmaceutical visit their earnings calendar on our site.</a>
This article was generated by Benzinga's automated content engine and reviewed by an editor.

Ultragenyx Pharmaceutical's Earnings: A Preview

This whale alert can help traders discover the next big trading opportunities.
Whales are entities with large sums of money and we track their transactions here at Benzinga on our options activity scanner.
Traders often look for circumstances when the market estimation of an option diverges away from its normal worth. Abnormal amounts of trading activity could push option prices to hyperbolic or underperforming levels. 
Below are some instances of options activity happening in the Health Care sector: 
<table>
<thead>
<tr>
<th>Symbol</th>
<th>PUT/CALL</th>
<th>Trade Type</th>
<th>Sentiment</th>
<th>Exp. Date</th>
<th>Strike Price</th>
<th>Total Trade Price</th>
<th align="right">Open Interest</th>
<th>Volume</th>
</tr>
</thead>
<tbody>
<tr>
<td>INO</td>
<td>CALL</td>
<td>SWEEP</td>
<td>BEARISH</td>
<td>11/21/25</td>
<td>$2.00</td>
<td>$30.8K</td>
<td align="right">332</td>
<td>4.0K</td>
</tr>
<tr>
<td>LLY</td>
<td>CALL</td>
<td>SWEEP</td>
<td>BEARISH</td>
<td>05/02/25</td>
<td>$820.00</td>
<td>$35.4K</td>
<td align="right">141</td>
<td>1.1K</td>
</tr>
<tr>
<td>RARE</td>
<td>CALL</td>
<td>SWEEP</td>
<td>BULLISH</td>
<td>08/15/25</td>
<td>$45.00</td>
<td>$44.3K</td>
<td align="right">255</td>
<td>1.0K</td>
</tr>
<tr>
<td>ABBV</td>
<td>CALL</td>
<td>SWEEP</td>
<td>BEARISH</td>
<td>05/16/25</td>
<td>$182.50</td>
<td>$28.8K</td>
<td align="right">780</td>
<td>752</td>
</tr>
<tr>
<td>LNTH</td>
<td>PUT</td>
<td>SWEEP</td>
<td>BEARISH</td>
<td>05/16/25</td>
<td>$100.00</td>
<td>$81.1K</td>
<td align="right">468</td>
<td>324</td>
</tr>
<tr>
<td>JNJ</td>
<td>CALL</td>
<td>TRADE</td>
<td>BEARISH</td>
<td>01/15/27</td>
<td>$165.00</td>
<td>$79.9K</td>
<td align="right">302</td>
<td>65</td>
</tr>
<tr>
<td>MRK</td>
<td>CALL</td>
<td>TRADE</td>
<td>BEARISH</td>
<td>06/18/26</td>
<td>$85.00</td>
<td>$34.1K</td>
<td align="right">236</td>
<td>59</td>
</tr>
<tr>
<td>PODD</td>
<td>PUT</td>
<td>SWEEP</td>
<td>BULLISH</td>
<td>01/16/26</td>
<td>$260.00</td>
<td>$35.7K</td>
<td align="right">5</td>
<td>10</td>
</tr>
</tbody>
</table>

<h2>Explanation</h2>
These bullet-by-bullet explanations have been constructed using the accompanying table. 
• Regarding INO (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/INO#NASDAQ">INO</a>), we observe a call option sweep with bearish sentiment. It expires in 204 day(s) on November 21, 2025. Parties traded 684 contract(s) at a $2.00 strike. This particular call needed to be split into 10 different trades to become filled. The total cost received by the writing party (or parties) was $30.8K, with a price of $45.0 per contract. There were 332 open contracts at this strike prior to today, and today 4000 contract(s) were bought and sold. 
• Regarding LLY (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/LLY#NYSE">LLY</a>), we observe a call option sweep with bearish sentiment. It expires in 1 day(s) on May 2, 2025. Parties traded 43 contract(s) at a $820.00 strike. This particular call needed to be split into 12 different trades to become filled. The total cost received by the writing party (or parties) was $35.4K, with a price of $836.0 per contract. There were 141 open contracts at this strike prior to today, and today 1130 contract(s) were bought and sold. 
• For RARE (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>), we notice a call option sweep that happens to be bullish, expiring in 106 day(s) on August 15, 2025. This event was a transfer of 80 contract(s) at a $45.00 strike. This particular call needed to be split into 24 different trades to become filled. The total cost received by the writing party (or parties) was $44.3K, with a price of $560.0 per contract. There were 255 open contracts at this strike prior to today, and today 1028 contract(s) were bought and sold. 
• For ABBV (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/ABBV#NYSE">ABBV</a>), we notice a call option sweep that happens to be bearish, expiring in 15 day(s) on May 16, 2025. This event was a transfer of 22 contract(s) at a $182.50 strike. This particular call needed to be split into 3 different trades to become filled. The total cost received by the writing party (or parties) was $28.8K, with a price of $1310.0 per contract. There were 780 open contracts at this strike prior to today, and today 752 contract(s) were bought and sold. 
• For LNTH (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/LNTH#NASDAQ">LNTH</a>), we notice a put option sweep that happens to be bearish, expiring in 15 day(s) on May 16, 2025. This event was a transfer of 163 contract(s) at a $100.00 strike. This particular put needed to be split into 53 different trades to become filled. The total cost received by the writing party (or parties) was $81.1K, with a price of $500.0 per contract. There were 468 open contracts at this strike prior to today, and today 324 contract(s) were bought and sold. 
• For JNJ (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/JNJ#NYSE">JNJ</a>), we notice a call option trade that happens to be bearish, expiring in 624 day(s) on January 15, 2027. This event was a transfer of 65 contract(s) at a $165.00 strike. The total cost received by the writing party (or parties) was $79.9K, with a price of $1230.0 per contract. There were 302 open contracts at this strike prior to today, and today 65 contract(s) were bought and sold. 
• For MRK (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/MRK#NYSE">MRK</a>), we notice a call option trade that happens to be bearish, expiring in 413 day(s) on June 18, 2026. This event was a transfer of 33 contract(s) at a $85.00 strike. The total cost received by the writing party (or parties) was $34.1K, with a price of $1035.0 per contract. There were 236 open contracts at this strike prior to today, and today 59 contract(s) were bought and sold. 
• For PODD (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PODD#NASDAQ">PODD</a>), we notice a put option sweep that happens to be bullish, expiring in 260 day(s) on January 16, 2026. This event was a transfer of 10 contract(s) at a $260.00 strike. This particular put needed to be split into 4 different trades to become filled. The total cost received by the writing party (or parties) was $35.7K, with a price of $3570.0 per contract. There were 5 open contracts at this strike prior to today, and today 10 contract(s) were bought and sold. 
Options Alert Terminology 
 - Call Contracts: The right to buy shares as indicated in the contract. 
 - Put Contracts: The right to sell shares as indicated in the contract. 
 - Expiration Date: When the contract expires. One must act on the contract by this date if one wants to use it. 
 - Premium/Option Price: The price of the contract. 
For more information, visit our <a href="https://pro.benzinga.com/blog/how-to-understand-option-alerts/">Guide to Understanding Options Alerts</a> or read <a href="https://www.benzinga.com/markets/options">more about unusual options activity</a>. 
This article was generated by Benzinga's automated content engine and reviewed by an editor.

Options

ABBV

LNTH

PODD

8 Health Care Stocks Whale Activity In Today's Session

JPMorgan on Thursday shared updated thoughts on Ultragenyx Pharmaceutical Inc&#8217;s (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) setrusumab program in <a href="https://www.benzinga.com/general/health-care" target="_blank" rel="noreferrer noopener">osteogenesis imperfecta (OI)</a>.
In a fourth-quarter 2024 press release, the company said patients continue dosing in the Phase 3&nbsp;Orbit&nbsp;and&nbsp;Cosmic&nbsp;trials, which evaluate setrusumab in pediatric and <a target="_blank" href="https://www.benzinga.com/pressreleases/25/02/g43711647/ultragenyx-reports-fourth-quarter-and-full-year-2024-financial-results-and-corporate-update" rel="noreferrer noopener">young adult patients with OI</a>.
The randomized, placebo-controlled Phase 3 portion of the&nbsp;Orbit&nbsp;study is progressing toward the second interim analysis in mid-2025 or a final analysis in the fourth quarter of 2025.
Also Read: <a target="_blank" href="https://www.benzinga.com/25/01/43012990/ultragenyx-stock-price-could-reach-70-analyst" rel="noreferrer noopener">Ultragenyx&#8217;s Multiple Commercial Assets And Candidates Impress Analyst; Sees Solid Upside</a>
Patients in the&nbsp;Cosmic&nbsp;study continue to be treated with either setrusumab or intravenous bisphosphonates (IV-BP) therapy and will be evaluated in parallel with the&nbsp;Orbit&nbsp;interim and final analyses.
JPMorgan analyst Anupam Rama said Thursday, &#8220;Ultragenyx remains on the J.P. Morgan US Equity Analyst Focus List.&#8221;
Rama maintains the Overweight rating and writes, &#8220;We believe Ultragenyx is uniquely positioned in the long term with a broad and diverse pipeline of orphan disease assets and a highly regarded management team with a strong track record in the space.&#8221;
The analyst believes the second interim analysis (IA2) of ORBIT has a much better chance of achieving statistical significance for the primary endpoint, the annualized fracture rate (AFR), compared to the first interim analysis (IA1).
If IA2 meets statistical significance, the analyst expects Ultragenyx stock to rise to around the mid-$60s to low-$70s per share. If it falls short, the stock could drop to the mid-$20s to low-$30s, making the potential reward outweigh the risk.
JPMorgan increased the <a href="https://www.benzinga.com/quote/RARE/analyst-ratings" target="_blank" rel="noreferrer noopener">price target from $104 to $117</a> based on the updated setrusumab revenue build, UX111 in MPS3A addition to the model, and expense adjustments.
In February, the U.S. Food and Drug Administration accepted for review the Biologics License Application seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A, with a Prescription Drug User Fee Act <a href="https://www.benzinga.com/pressreleases/25/02/g43779022/ultragenyx-announces-fda-acceptance-and-priority-review-of-the-biologics-license-application-bla-f" target="_blank" rel="noreferrer noopener">action date of Aug. 18</a>.
Price Action: RARE stock is up 2.6% at $38.98 at last check Thursday.
Read Next:
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<li><a href="https://www.benzinga.com/25/03/44519562/nvidia-faces-fresh-china-setback-as-chip-shortages-and-sanctions-disrupt-supply" target="_blank" rel="noreferrer noopener">Nvidia Faces Fresh China Setback As Chip Shortages And Sanctions Disrupt Supply</a></li>
</ul>
Photo: Shutterstock

Ultragenyx Stays On JPMorgan's Focus List As Analyst Highlights Strong Long-Term Positioning

In the preceding three months, 10 analysts have released ratings for Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>), presenting a wide array of perspectives from bullish to bearish.
The following table provides a quick overview of their recent ratings, highlighting the changing sentiments over the past 30 days and comparing them to the preceding months.
<table>
<thead>
<tr>
<th></th>
<th align="right">Bullish</th>
<th align="right">Somewhat Bullish</th>
<th align="right">Indifferent</th>
<th align="right">Somewhat Bearish</th>
<th align="right">Bearish</th>
</tr>
</thead>
<tbody>
<tr>
<td>Total Ratings</td>
<td align="right">4</td>
<td align="right">5</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>Last 30D</td>
<td align="right">0</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>1M Ago</td>
<td align="right">2</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>2M Ago</td>
<td align="right">1</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>3M Ago</td>
<td align="right">1</td>
<td align="right">2</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
</tbody>
</table>
Analysts have recently evaluated Ultragenyx Pharmaceutical and provided 12-month price targets. The average target is $104.8, accompanied by a high estimate of $140.00 and a low estimate of $48.00. This current average has increased by 0.96% from the previous average price target of $103.80. 
<img src="https://www.benzinga.com/files/images/story/2025/1742248820_0.png" alt="price target chart" />
<h2>Investigating Analyst Ratings: An Elaborate Study</h2>
In examining recent analyst actions, we gain insights into how financial experts perceive Ultragenyx Pharmaceutical. The following summary outlines key analysts, their recent evaluations, and adjustments to ratings and price targets.
<table>
<thead>
<tr>
<th>Analyst</th>
<th>Analyst Firm</th>
<th>Action Taken</th>
<th>Rating</th>
<th>Current Price Target</th>
<th>Prior Price Target</th>
</tr>
</thead>
<tbody>
<tr>
<td>Allison Bratzel</td>
<td>Piper Sandler</td>
<td>Lowers</td>
<td>Overweight</td>
<td>$115.00</td>
<td>$140.00</td>
</tr>
<tr>
<td>Kristen Kluska</td>
<td>Cantor Fitzgerald</td>
<td>Maintains</td>
<td>Overweight</td>
<td>$118.00</td>
<td>$118.00</td>
</tr>
<tr>
<td>Ed Arce</td>
<td>HC Wainwright &amp; Co.</td>
<td>Maintains</td>
<td>Buy</td>
<td>$95.00</td>
<td>$95.00</td>
</tr>
<tr>
<td>Whitney Ijem</td>
<td>Canaccord Genuity</td>
<td>Raises</td>
<td>Buy</td>
<td>$136.00</td>
<td>$121.00</td>
</tr>
<tr>
<td>Ed Arce</td>
<td>HC Wainwright &amp; Co.</td>
<td>Maintains</td>
<td>Buy</td>
<td>$95.00</td>
<td>$95.00</td>
</tr>
<tr>
<td>Kristen Kluska</td>
<td>Cantor Fitzgerald</td>
<td>Maintains</td>
<td>Overweight</td>
<td>$118.00</td>
<td>$118.00</td>
</tr>
<tr>
<td>Christopher Raymond</td>
<td>Piper Sandler</td>
<td>Raises</td>
<td>Overweight</td>
<td>$140.00</td>
<td>$135.00</td>
</tr>
<tr>
<td>Laura Chico</td>
<td>Wedbush</td>
<td>Maintains</td>
<td>Neutral</td>
<td>$48.00</td>
<td>$46.00</td>
</tr>
<tr>
<td>Ed Arce</td>
<td>HC Wainwright &amp; Co.</td>
<td>Maintains</td>
<td>Buy</td>
<td>$95.00</td>
<td>$95.00</td>
</tr>
<tr>
<td>Tiago Fauth</td>
<td>Wells Fargo</td>
<td>Raises</td>
<td>Overweight</td>
<td>$88.00</td>
<td>$75.00</td>
</tr>
</tbody>
</table>
<h3>Key Insights:</h3>
<ul>
<li>Action Taken: Analysts frequently update their recommendations based on evolving market conditions and company performance. Whether they 'Maintain', 'Raise' or 'Lower' their stance, it reflects their reaction to recent developments related to Ultragenyx Pharmaceutical. This information provides a snapshot of how analysts perceive the current state of the company.</li>
<li>Rating: Analyzing trends, analysts offer qualitative evaluations, ranging from 'Outperform' to 'Underperform'. These ratings convey expectations for the relative performance of Ultragenyx Pharmaceutical compared to the broader market.</li>
<li>Price Targets: Gaining insights, analysts provide estimates for the future value of Ultragenyx Pharmaceutical's stock. This comparison reveals trends in analysts' expectations over time.</li>
</ul>
Assessing these analyst evaluations alongside crucial financial indicators can provide a comprehensive overview of Ultragenyx Pharmaceutical's market position. Stay informed and make well-judged decisions with the assistance of our Ratings Table.
<a href="https://www.benzinga.com/quote/RARE/analyst-ratings?adType=benzinga-insights&amp;ad=analyst-ratings&amp;campaign=wallstreetadvantage">Stay up to date on Ultragenyx Pharmaceutical analyst ratings.</a>
<h2>Discovering Ultragenyx Pharmaceutical: A Closer Look</h2>
Ultragenyx Pharmaceutical Inc is a USA-based biopharmaceutical company. It identifies, acquires, develops, and commercializes novel products for the treatment of serious rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. The company's medicine portfolio includes Crysvita, Dojolvi and Mepsevii. Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 year of age and older. Mepsevii is indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII.
<h3>A Deep Dive into Ultragenyx Pharmaceutical's Financials</h3>
Market Capitalization: With restricted market capitalization, the company is positioned below industry averages. This reflects a smaller scale relative to peers.
Revenue Growth: Ultragenyx Pharmaceutical's remarkable performance in 3 months is evident. As of 31 December, 2024, the company achieved an impressive revenue growth rate of 29.42%. This signifies a substantial increase in the company's top-line earnings. When compared to others in the Health Care sector, the company faces challenges, achieving a growth rate lower than the average among peers.
Net Margin: The company's net margin is a standout performer, exceeding industry averages. With an impressive net margin of -80.9%, the company showcases strong profitability and effective cost control.
Return on Equity (ROE): Ultragenyx Pharmaceutical's ROE is below industry averages, indicating potential challenges in efficiently utilizing equity capital. With an ROE of -44.3%, the company may face hurdles in achieving optimal financial returns.
Return on Assets (ROA): Ultragenyx Pharmaceutical's ROA is below industry averages, indicating potential challenges in efficiently utilizing assets. With an ROA of -8.77%, the company may face hurdles in achieving optimal financial returns.
Debt Management: Ultragenyx Pharmaceutical's debt-to-equity ratio is below industry norms, indicating a sound financial structure with a ratio of 0.16.
<h2>Analyst Ratings: What Are They?</h2>
Analysts are specialists within banking and financial systems that typically report for specific stocks or within defined sectors. These people research company financial statements, sit in conference calls and meetings, and speak with relevant insiders to determine what are known as analyst ratings for stocks. Typically, analysts will rate each stock once a quarter. 
Some analysts also offer predictions for helpful metrics such as earnings, revenue, and growth estimates to provide further guidance as to what to do with certain tickers. It is important to keep in mind that while stock and sector analysts are specialists, they are also human and can only forecast their beliefs to traders.
Which Stocks Are Analysts Recommending Now?
Benzinga Edge gives you instant access to all major analyst upgrades, downgrades, and price targets. Sort by accuracy, upside potential, and more. <a href="https://www.benzinga.com/premium/ideas/benzinga-edge-checkout/?t=be8be9arja3be22bz21&amp;adType=benzinga-insights&amp;ad=analyst-ratings&amp;campaign=wallstreetadvantage">Click here to stay ahead of the market</a>.
This article was generated by Benzinga's automated content engine and reviewed by an editor.

Demystifying Ultragenyx Pharmaceutical: Insights From 10 Analyst Reviews

Piper Sandler analyst Allison Bratzel maintains Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) with a Overweight and lowers the price target from $140 to $115.

Piper Sandler Maintains Overweight on Ultragenyx Pharmaceutical, Lowers Price Target to $115

Cantor Fitzgerald analyst Kristen Kluska reiterates Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) with a Overweight and maintains $118 price target.

Cantor Fitzgerald Reiterates Overweight on Ultragenyx Pharmaceutical, Maintains $118 Price Target

HC Wainwright &amp; Co. analyst Ed Arce reiterates Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) with a Buy and maintains $95 price target.

HC Wainwright & Co. Reiterates Buy on Ultragenyx Pharmaceutical, Maintains $95 Price Target

Ultragenyx Pharmaceutical Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) today announced the U.S. Food and Drug Administration (FDA or the Agency) has accepted for review the Biologics License Application (BLA) seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). The FDA granted the BLA Priority Review with a Prescription Drug User Fee Act (PDUFA) action date of August 18, 2025. The FDA also informed the company that they are not currently planning to hold an advisory committee meeting to discuss this application.

Ultragenyx Pharmaceutical Announces FDA Acceptance And Priority Review Of The BLA For UX111 AAV Gene Therapy To Treat Sanfilippo Syndrome Type A

Press Releases

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

Ultragenyx to Host Conference Call for First Quarter 2025 Financial Results and Corporate Update

Ultragenyx Reports First Quarter 2025 Financial Results and Corporate Update

Ultragenyx to Participate at Bank of America's 2025 Healthcare Conference

Ultragenyx Underscores Continued Commitment to Rare Disease Innovation with 2024 Impact Report

Ultragenyx to Participate at Goldman Sachs 46th Annual Global Healthcare Conference

Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome

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