Latest news with #UltragenyxPharmaceuticalInc
Yahoo
14-07-2025
- Health
- Yahoo
Health Canada Extends the Approval of Evkeeza® (evinacumab) to Children as Young as 6-months Old with Homozygous Familial Hypercholesterolemia (HoFH)
First and only medicine approved by Health Canada for patients as young as 6-months of age with HoFH, an ultrarare, inherited form of high cholesterol TORONTO, July 14, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultra-rare genetic diseases, today announced that Health Canada has extended the approval of Evkeeza® (evinacumab) as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies to treat children aged 6-months and older with homozygous familial hypercholesterolemia (HoFH). Evkeeza, an angiopoietin-like 3 (ANGPTL3) inhibitor initially received Health Canada approval in September 2023 as an adjunct to diet and other LDL-C lowering therapies for the treatment of adult and pediatric patients aged 5 years and older with HoFH, a disease associated with dangerously high levels of LDL-C. "Early detection and prompt initiation of effective treatment are essential to prevent the devastating consequences of very high cholesterol that begins at a very young age for patients with HoFH. However, conventional treatment options are challenging for infants and children,' stated Dr. Brian McCrindle, a pediatric lipid specialist at The Hospital for Sick Children, University of Toronto. 'This extended approval will enable the very early and effective lowering of lipid levels essential for these vulnerable patients." The pharmacokinetics and efficacy of the drug in pediatric patients aged 6 months to less than 5 years with HoFH have been predicted from a model-based extrapolation analysis. Results of these analyses show that pediatric patients aged 6 months to less than 5 years are predicted to experience a similar or higher magnitude of percent change in LDL-C at week 24 compared to older pediatric patients and adults, when receiving a 15 mg/kg dose every 4 weeks. In addition, supportive data for five patients who initiated treatment between 1 and 4 years of age via compassionate use showed a clinically meaningful reduction of LDL-C consistent with that observed in patients aged 5 years or older in clinical studies. Based on the currently available data, the safety profile in pediatric patients aged 6-months to 5 years old is expected to be similar to the safety profile in older pediatric patients. No new safety concerns have been identified in the compassionate use program. "HoFH is a severe, life-threatening condition that, if left untreated, can lead to heart disease and death as early as childhood. This makes diagnosing it and reducing the resulting high LDL-C levels an urgent need,' said Monty Keast, Vice President and General Manager at Ultragenyx Canada. 'We are proud to be able to provide children as young as 6-months old living with HoFH a medicine that could reduce LDL-C levels and will work collaboratively with healthcare providers and payers across the country to ensure it is accessible for families.' Evkeeza is reimbursed and commercially available to prescribe for appropriate patients with HoFH in Canada via private drug plans and through the public drug program in Quebec, the UK, U.S., Italy, Japan, the Netherlands, Spain and Luxembourg. It is also available via early access programs in 13 additional countries including Austria and France. About Homozygous Familial Hypercholesterolemia (HoFH)HoFH is a devastating form of inherited hypercholesterolemia, affecting 1 in 300,000 people globally and 1 in 275,000 in the French Canadian population. HoFH occurs when two copies of the familial hypercholesterolemia (FH)-causing genes are inherited, one from each parent, resulting in dangerously high levels (>10 mmol/L) of LDL-C, or bad cholesterol. Patients with HoFH are at risk for premature atherosclerotic disease and cardiac events at an early age. For more information about HoFH please go to About Evkeeza (evinacumab)Evinacumab, the active substance in the medicine, attaches to a protein in the body called ANGPTL3 and blocks its effects. ANGPTL3 is involved in controlling cholesterol levels and blocking its effect reduces the level of cholesterol in the blood. The medicine is delivered via an infusion every month (4 weeks). Regeneron Pharmaceuticals, Inc. discovered and developed Evkeeza, and commercializes the product in HoFH in the U.S. under the generic name evinacumab-dgnb, with dgnb as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the FDA. Ultragenyx is responsible for development and commercialization efforts in countries outside of the U.S. What is Evkeeza used for? Evkeeza is used to treat adults and children aged 6 months and older with very high cholesterol caused by a condition called homozygous familial hypercholesterolemia (HoFH). Evkeeza is used with a low-fat diet and other medicines to bring down cholesterol levels. Do not use Evkeeza if: • Allergic to evinacumab or to any of the ingredients in this medicine. To help avoid side effects and ensure proper use, a healthcare professional should be consulted before taking Evkeeza. Talk about any health conditions or problems, including: • Allergic reactions (hypersensitivity), including a severe reaction known as anaphylaxis. Symptoms may include swelling of the lips, tongue, or throat that make it difficult to swallow or breathe, and may also include wheezing, feeling dizzy, or fainting. If any of these symptoms are noticed, stop taking Evkeeza and get immediate medical help. • If pregnant, might be pregnant, or plan to become pregnant, ask a healthcare professional for advice before taking Evkeeza. Evkeeza may harm an unborn baby. Tell a healthcare professional if become pregnant while using Evkeeza. • If breastfeeding or plan to breastfeed, ask a healthcare professional for advice before being given Evkeeza. It is not known if Evkeeza passes into breast milk. Tell a healthcare professional about all the medicines taken, including any drugs, vitamins, minerals, natural supplements, or alternative medicines. What are the possible side effects from using Evkeeza? These are not all the possible side effects when taking Evkeeza. If any side effects not listed here are experienced, tell a healthcare professional. • Abdominal pain • Back pain • Constipation • Decreased energy • Dizziness • Flu symptoms • Itchiness at the site of the injection • Nausea • Pain in legs or arms • Runny nose • Fatigue (for patients aged 5-11 years old) • Sore throat or sinus infection Reporting Side Effects: Any suspected side effects associated with the use of health products can be reported to Health Canada by: • Visiting the Web page on Adverse Reaction Reporting ( for information on how to report online, by mail or by fax; or • Calling toll-free at 1-866-234-2345. NOTE: Contact a health professional if information is needed about how to manage side effects. The Canada Vigilance Program does not provide medical advice. Please see PRODUCT MONOGRAPH INCLUDING PATIENT MEDICATION INFORMATION for more information. About Ultragenyx Pharmaceutical is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has 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. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit the company's website at: Ultragenyx Forward-Looking Statements and Use of Digital MediaExcept for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause the Company's clinical development programs, commercial success of its products and product candidates, continued collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, risks related to reliance on third party partners to conduct certain activities on the Company's behalf; the potential for any license or collaboration agreement, including Ultragenyx's collaboration agreement with Regeneron to commercialize Evkeeza outside of the United States, to be terminated; uncertainty and potential delays related to clinical drug development; uncertainties and unpredictability of obtaining regulatory approval for the Company's product candidates and the scope of such potential regulatory approval; smaller than anticipated market opportunities for the Company's products and product candidates; fluctuations in buying or distribution patterns by distributors and specialty pharmacies; competition to the Company's products and product candidates; potential undesirable or serious side effects from the Company's products or product candidates; the Company's ability to effectively manage the expansion of its commercial organization; market acceptance of the Company's current or future products; uncertainties related to insurance coverage and reimbursement status of newly approved products; manufacturing risks and supply chain disruptions; competition from other therapies or products; and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the Company's future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx's products and product candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 7, 2025, and its subsequent periodic reports filed with the SEC. In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx's Investor Relations website ( and LinkedIn website ( ContactsUltragenyx Pharmaceutical Higa+1-415-475-6370ir@ MediaJoey Fleury+1-925-784-5829media@
Yahoo
03-06-2025
- Business
- Yahoo
Ultragenyx to Participate at Goldman Sachs 46th Annual Global Healthcare Conference
NOVATO, Calif., June 03, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil Kakkis, M.D., Ph.D., the company's chief executive officer, will participate in a fireside at the Goldman Sachs 46th Annual Global Healthcare Conference on Tuesday, June 10, 2025, at 10:00 a.m. ET. The live and archived webcast of the panel will be accessible from the company's website at About Ultragenyx Pharmaceutical Inc. Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has 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. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit the company's website at: Contacts – Ultragenyx Pharmaceutical, Higa ir@
Yahoo
03-06-2025
- Business
- Yahoo
Ultragenyx to Participate at Goldman Sachs 46th Annual Global Healthcare Conference
NOVATO, Calif., June 03, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil Kakkis, M.D., Ph.D., the company's chief executive officer, will participate in a fireside at the Goldman Sachs 46th Annual Global Healthcare Conference on Tuesday, June 10, 2025, at 10:00 a.m. ET. The live and archived webcast of the panel will be accessible from the company's website at About Ultragenyx Pharmaceutical Inc. Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has 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. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit the company's website at: Contacts – Ultragenyx Pharmaceutical, Higa ir@
Yahoo
14-05-2025
- Business
- Yahoo
Ultragenyx Underscores Continued Commitment to Rare Disease Innovation with 2024 Impact Report
NOVATO, Calif., May 14, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) released its 2024 Impact Report today, which covers the company's approach to corporate responsibility. The report highlights efforts to transform the lives of individuals and families affected by rare diseases, empower its employees, and make a meaningful impact on the communities it operates within and serves. 'Since founding Ultragenyx 15 years ago, I've sought to build a next-generation rare disease company that has a meaningful and sustained impact on the entire rare disease community,' said Emil Kakkis, M.D., Ph.D., founder, president, and chief executive officer of Ultragenyx. 'Guided by the principle of doing the right thing, we have engineered our own dynamic approach to drug development while working on broader education and policy initiatives that meaningfully support diagnosis, drug development, regulatory pathways, and clinical care. The sustainability and social impact implications of our actions and decisions are consistently considered and reflected in our 2024 Impact Report.' The company's approach to corporate responsibility is structured around six key pillars—Innovation, Patients, People, Communities, Planet, and Governance—aligned with its vision to lead the future of rare disease medicine and its mission to transform the lives of people with rare diseases. The 2024 Ultragenyx Impact Report features updates for all six pillars, as well as Sustainability Accounting Standards Board (SASB) and Global Reporting Initiative (GRI) indices. For the full report and more on Ultragenyx's Corporate Responsibility efforts, visit Highlights from the company's 2024 report include: Innovation:Ultragenyx is committed to developing and delivering transformative treatments where none exist. Successfully transferred the manufacturing process of our DTX401 gene therapy to our Gene Therapy Manufacturing Facility (GTMF) in Bedford, Massachusetts Advanced several key clinical programs to treat rare and ultra-rare diseases, including osteogenesis imperfecta (OI), Angelman syndrome, Sanfilippo syndrome type A (MPS IIIA), and glycogen storage disease type Ia (GSDIa) Supported more than 80 clinical and non-clinical investigator-sponsored trials (ISTs) globally Patients:Ultragenyx is committed to supporting the rare disease community through our efforts to develop novel therapies, share our science and expertise, achieve broad access to screening and treatment, and partner with policymakers for meaningful change. Supported more than 650 patients in 50 countries in gaining access to Ultragenyx treatments through various global expanded access and patient assistance programs since 2013 Successfully advocated for the passage of newborn screening legislation in two more U.S. states Hosted the ninth and tenth Rare Bootcamp to help patient families, foundations, and organizations seeking to develop novel treatments for rare diseases People:Ultragenyx is committed to maintaining an inclusive, safe, and healthy environment. The company is also committed to fair and equitable compensation practices that are transparent and free from bias. Launched the Executive Edge program to support the development of leadership skills at the vice president (VP) and senior VP levels Maintained a high engagement score of 88% in our employee engagement survey Communities:Ultragenyx is committed to supporting initiatives that provide meaningful impact for the rare disease community: public health and access to care, Science, Technology, Engineering, Arts, and Mathematics (STEAM) education, and local, at-risk communities. Approved more than $3 million in charitable donations, independent medical education, and health-related grants across more than 20 countries Hosted 2nd Annual Ultragenyx Global Days of Service, with employees volunteering in nearly 60 service projects Planet:Ultragenyx is committed to implementing an environmental strategy that helps minimize its environmental footprint across its business. Purchased 100% renewable electricity for our corporate headquarters campus in Novato, Calif. Achieved My Green Lab Green certification at our Translational Sciences lab in Novato, Calif. Governance:Ultragenyx is committed to strong corporate governance, ethics and integrity, compliance, data protection and security, and responsible procurement. More than 90% of responders to the annual compliance culture survey stated that the annual compliance training is adequate for them to confidently execute their responsibilities Maintained a formal Global Human Rights policy to hold ourselves and our vendors to its standards Ultragenyx Receives External Recognition in 2024Ultragenyx's achievements in 2024 were consistently recognized through several prestigious awards. In 2024, Ultragenyx was named one of the 100 Most Sustainable Companies by Barron's and one of the Top Places to Work in the USA for the second consecutive year, receiving six Cultural Excellence Awards from Top Workplaces. The company also received accolades from The Boston Globe and the San Francisco Chronicle. Additionally, the company was honored with the MassEcon Gold Award for Economic Impact in the Northeast region, celebrating its significant contributions to the Massachusetts economy, and was recognized as one of the Best Places for Working Parents in Massachusetts. About UltragenyxUltragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has 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. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit the company's website at: Forward-Looking Statements and Use of Digital Media This press release and the 2024 Ultragenyx Impact Report and other materials cross-referenced in this press release contain statements that are aspirational or reflective of our views about the company's future performance that constitute 'forward looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are generally identified through the inclusion of words such as 'aim,' 'anticipate,' 'aspire,' 'believe,' commit,' 'endeavor,' 'estimate,' 'expect,' 'goal,' 'intend,' 'may,' 'plan,' 'seek,' 'strive,' 'target,' 'will,' vision,' 'mission,' 'strategy,' 'commitment' and 'work,' or similar statements or variations of such terms and other similar expressions. The forward-looking statements in this document and the materials cross-referenced concern Ultragenyx's goals, progress or expectations with respect to corporate responsibility, sustainability, patients, products, product candidates, employees, environmental matters, policy and business risks and opportunities. Forward-looking statements inherently involve risks and uncertainties that could cause actual results to differ materially from those predicted in such statements. These statements are based on numerous assumptions that the company believes are reasonable but are open to a wide range of uncertainties and business risks. In addition, these statements may be based on standards for measuring progress that are still developing, controls and processes that continue to evolve, and assumptions that are subject to change in the future, and certifications, representations or data reviewed or provided by third parties Consequently, actual results may vary materially from what is contained in a forward-looking statement. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 7, 2025, and its subsequent periodic reports filed with the SEC. Forward-looking statements are aspirational and are not guarantees or promises that goals or targets will be met. Ultragenyx undertakes no obligation to update any forward-looking or other statements, whether as a result of new information, future events, or otherwise, and notwithstanding any historical practice of doing so. Ultragenyx may determine to adjust any goals and targets or establish new ones to reflect changes in its business. The information included in, and any issues identified as material for purposes of, the 2024 Ultragenyx Impact Report is not an indication that they are considered material to Ultragenyx, its investors or other stakeholders, or required to be disclosed in the company's filings, in each case under SEC reporting or any other laws or requirements that may apply to the company. In the context of this report, the term 'material' is distinct from, and should not be confused with, such term as defined for SEC or other mandatory reporting purposes. In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx's Investor Relations website ( and LinkedIn website ( Ultragenyx Contacts Investors Joshua Higa(415) 475-6370ir@ MediaJoey Fleurymedia@
Yahoo
30-04-2025
- Business
- Yahoo
Ultragenyx to Host Conference Call for First Quarter 2025 Financial Results and Corporate Update
NOVATO, Calif., April 30, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Tuesday, May 6, 2025, to discuss its financial results and corporate update for the quarter ending March 31, 2025. The live and replayed webcast of the call will be available through the company's website at The replay of the call will be available for three months. About Ultragenyx Pharmaceutical is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has 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. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit the company's website at: Contact Ultragenyx InvestorsJoshua Higa(415) 475-6370ir@ MediaJoey Fleury (415) 946-1090media@ in to access your portfolio
