First and only medicine approved in the EU for paediatric patients aged 6-months to 5 years old with HoFH, an ultrarare, inherited form of high cholesterolBASEL, Switzerland, Jan. 06, 2025 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultrarare genetic diseases, today announced that the European Commission (EC) has extended the approval of Evkeeza® (evinacumab) as an adjunct to diet and other lipid-lowering therapies to treat children aged 6-months and older with homozygous familial hypercholesterolemia (HoFH). Evkeeza, an angiopoietin-like 3 (ANGPTL3) inhibitor, is the first medicine indicated for children in the European Union (EU) as young as 6-months old to treat HoFH, a disease associated with dangerously high levels of low-density lipoprotein cholesterol (LDL-C). “For very young children suffering from homozygous HoFH, leading to dangerously high LDL-C levels and early cardiovascular disease, treatment options are limited. Many young children don’t reach treatment goals, leaving them with an uncertain future,” stated Albert Wiegman, M.D., Ph.D. and Professor, Department of Paediatrics at Amsterdam University Medical Center. “Evinacumab has demonstrated significant LDL-C reductions in adults, adolescents, and children with HoFH from 5-years onwards. The current label expansion for children younger than 5-years illustrates the potential of this medicine to help them control their LDL-C levels at even earlier age in the course of their disease.” This EC decision follows the positive recommendation received from the Committee for Medicinal Products for Human Use in November 2024.The efficacy of Evkeeza in paediatric patients aged 6 months to less than 5 years with HoFH has been predicted from a model-based extrapolation analysis. Results of these analyses show that paediatric 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 adults, when receiving a 15 mg/kg dose every 4 weeks. In addition, supportive data for five patients between 1 and 4 years old with HoFH who received Evkeeza via compassionate use show 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.   “The international community of parents and caregivers of children with HoFH welcomes this approval, because this is a potentially life-changing therapy for the children and their parents affected by this rare and severe disorder,” stated Magdalena Daccord, chief executive officer of FH Europe Foundation. “As we advocate for childhood screening and detection to help improve early HoFH diagnosis, it is key to be able to offer to young patients appropriate and innovative treatment solutions along with lifestyle management. That said, it will be a true success once this therapy option is available to all those who need it as soon as they need it.” Evkeeza initially received approval as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C)-lowering therapies for the treatment of adult and adolescent patients aged 12 years and older with HoFH in June 2021. The indication was later extended to include children aged 5 to 11 years old with HoFH, with EC decision for that variation received in December 2023. The treatment is now reimbursed and commercially available to prescribe for appropriate patients with HoFH in the UK, U.S., Canada, Italy, Japan, the Netherlands, Spain and Luxembourg. It is also available via early access schemes in 13 additional countries including Austria and France. “HoFH can cause severe cardiovascular events including heart attacks even in young children, making diagnosing it and reducing the high LDL-C levels it causes an urgent matter,” said Jane Cooper, senior vice president, EMEA region head at Ultragenyx. “We are proud to be able to provide children as young as 6-months old living with HoFH the first approved medicine to reduce LDL-C levels and hope that it will support a fundamental shift in the management of the disease.” About Homozygous Familial Hypercholesterolemia (HoFH)HoFH is a devastating form of inherited hypercholesterolemia, affecting 1 in 300,000 people globally and approximately 1,600 people in the European Union. HoFH occurs when two copies of the familial hypercholesterolemia (FH)-causing genes are inherited, one from each parent, resulting in dangerously high levels ( >400 mg/dL) of LDL-C, or bad cholesterol. Patients with HoFH are at risk for premature atherosclerotic disease and cardiac events at an early age. About Evkeeza (evinacumab)Evinacumab, the active substance in Evkeeza, 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. Evkeeza is delivered via an infusion every month (4 weeks). Evkeeza is approved by the European Commission (EC) as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies for the treatment of adult and paediatric patients aged 6-months and older with homozygous familial hypercholesterolemia (HoFH). 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 for Evkeeza in countries outside of the U.S. IMPORTANT SAFETY INFORMATION FOR EVKEEZA (evinacumab)The most common side effects ( >10%) include symptoms of the common cold, such as runny nose (nasopharyngitis) and for children below the age of 11 years old feeling tired (fatigue). Evkeeza can cause serious allergic reactions. Tell your doctor or nurse immediately if you get any symptoms of a severe allergic reaction: swelling – mainly of the lips, tongue or throat, which makes it difficult to swallow or breathe, breathing problems or wheezing, feeling dizzy or fainting, rash, hives, itching. The drip will be stopped immediately, and you may need to take other medicines to control the reaction. Who should not use EVKEEZA (evinacumab)?You should not be given Evkeeza if you are allergic to evinacumab or any of the other ingredients of this medicine. Evkeeza is not recommended for children below the age of 6 months because there is not yet enough information on its use in this group of patients.Tell your doctor if you are taking, have recently taken or might take any other medicines.If you are pregnant, think you may be pregnant or are planning to have a baby, ask your doctor for advice before taking this medicine. Evkeeza may harm your unborn baby. Tell your doctor immediately if you become pregnant while you are being treated with Evkeeza. If you are able to become pregnant, you should use effective contraception to avoid becoming pregnant. Use effective contraception while you are being treated with Evkeeza and use effective contraception for at least 5 months after the last dose of Evkeeza. Talk to your doctor about the best contraception method for you during this time. If you are breast-feeding or plan to breast-feed, ask your doctor for advice before you are given this medicine. It is not known if Evkeeza passes into breast milk. If you get any side effects, talk to your doctor or nurse. By reporting side effects, you can help provide more information on the safety of this medicine. You may report side effects via email to ultragenyx@primevigilance.com. Please see full Product Information. About Ultragenyx Pharmaceutical Inc.Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultrarare 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: www.ultragenyx.com. 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 November 6, 2024, 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 (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/). ContactsUltragenyx Pharmaceutical Inc. InvestorsJoshua Higa+1-415-475-6370ir@ultragenyx.com MediaJoey Fleury+1-925-784-5829media@ultragenyx.com