Hyperlipidemia Type IIa

FERNANDINA BEACH, Fla.--( BUSINESS WIRE )--A new study published in Circulation: Cardiovascular Quality and Outcomes from the Family Heart Foundation — a patient-centered research and advocacy nonprofit organization dedicated to improving the lives of families impacted by inherited lipid disorders and LDL-cholesterol — revealed that utilization of proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9i) to reduce low-density lipoprotein cholesterol (LDL-C) remains low among high-risk patients. Despite key developments between 2017 and early 2019 that were expected to increase appropriate utilization, rejection of prescribed PCSK9i by insurance plans remains uncharacteristically high compared to other similar therapies for cardiovascular and metabolic disease. Since 2019, PCSK9i have had a label expansion; positive results from major outcomes trials and large studies showing the use of PCSK9 inhibitors reducing cardiac events; a 60% price reduction on PCSK9i and established clinical practice guidelines all of which were supposed to improve utilization, yet access is still a barrier leaving patients at risk for heart attacks and strokes. The burden of cardiovascular disease, driven largely by atherosclerosis, is increasing in the United States. High LDL-C is a major modifiable risk factor. A 2019 study by the Family Heart Foundation (formerly known as the FH Foundation) published in Circulation: Cardiovascular Quality and Outcomes found that compared to individuals who were able to obtain their prescribed PCSK9i, those whose prescription was rejected by insurance plans or abandoned experienced significantly more heart attacks, strokes, and other cardiovascular events within 12 months. “The results from this new Family Heart Foundation study suggest that patients still experience substantial challenges getting the PCSK9i that have been prescribed for them by their medical team, despite guidelines recommending their use and extensive evidence documenting their role in LDL-C reduction and the prevention of heart attack or stroke," said Diane E. MacDougall, Vice President of Science and Research at the Family Heart Foundation and co-author of the study. “As a result, eligible patients remain at higher risk of heart attacks and other major cardiovascular events as demonstrated by the 2019 study.” Key findings from the current study showed continued barriers for PCSK9i use: Despite improving since 2018, 30.95% of PCSK9i prescriptions are rejected by insurance plans. This is significantly higher compared to other guideline-recommended cardiometabolic therapies with demonstrated cardiovascular benefit (rejection rates range from 3.53% to 14.61%). Despite developments that were expected to increase PCSK9i utilization, new PCSK9i prescriptions remained low, at 470,018 during the 2019-2021 timeframe compared with 238,704 during the 2015-2018 timeframe. Taking into account both rejections by insurance plans and abandonments, paid prescription rate for PCSK9i coverage was substantially lower (49.93%) than those for other guideline-recommended cardiometabolic therapies (ranging from 68.49% to 84.45%). While statins are the first line of treatment, they may not lower LDL-C enough for high-risk patients. The 2018 ACC/AHA Multi-society Guidelines on the Management of Blood Cholesterol recommended the use of PCSK9i for appropriate patients. PCSK9i were approved by the U.S. Food & Drug Administration in 2015 as a major advancement to lower elevated LDL-C in patients with ASCVD and familial hypercholesterolemia. After 2017, the label for PCSK9i was expanded to include reduction of cardiovascular events in ASCVD patients and reduction of elevated LDL-C in patients with primary hypercholesterolemia, based on positive data from the FOURIER and ODYSSEY Outcomes trials. In addition, PCSK9i have been similarly priced to other guideline-indicated cardiometabolic drugs since 2018. About the Study Titled, “Trends in Patient Access to, and Utilization of, Prescribed PCSK9 Inhibitors in a Large U.S. Claims Database from 2015 to 2021,” the retrospective study was based on anonymized data from the Family Heart Database™ comprised of U.S. healthcare claims data representing more than half of the U.S. census. It analyzed a cohort of adults prescribed a PCSK9i (evolocumab or alirocumab) and other branded guideline-recommended, cardiometabolic medications comparing 2015-2018 and 2019-2021. Patients’ coverage for new prescriptions of PCSK9i and cardiometabolic medications was classified as: paid (covered by insurer, retrieved by individual); rejected (rejected by insurer); or abandoned (covered by insurer, not retrieved). About the Family Heart Foundation The Family Heart Foundation is a nonprofit patient-centered research and advocacy organization dedicated to improving the lives of families impacted by inherited lipid disorders. The Foundation is a pioneer in the application of real-world evidence, patient-driven advocacy, and multi-stakeholder education to help prevent heart attacks and strokes caused by familial hypercholesterolemia (FH) and elevated Lipoprotein(a), or Lp(a), two common genetic disorders that have an impact across generations. The Family Heart Foundation conducts innovative research to break down barriers to diagnosis and management of inherited lipid disorders; educates patients, providers, and policy makers; advocates for change; and provides hope and support for families impacted by heart disease and stroke caused by FH, HoFH, and high Lp(a). The organization was founded in 2011 as the FH Foundation. For more information, visit FamilyHeart.org and follow us on Twitter , Facebook , Instagram and LinkedIn .

NOVATO, Calif., Jan. 04, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending Evkeeza® (evinacumab) to NHS England. Evkeeza is recommended within its marketing authorization as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adults and adolescent patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH). Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for this rare and debilitating condition. "We are very pleased that NICE has approved the use of evinacumab within the NHS. Our patients with HoFH often have early onset heart disease, in many cases in their teens, and many of the usual medicines we use to treat cholesterol have limited effectiveness in this condition,” stated Dr. Jaimini Cegla, clinical lead of the Lipid and Cardiovascular Risk Service, Hammersmith Hospital. “Evinacumab, which is effective at lowering LDL-C in HoFH when combined with other lipid-lowering therapies, is a much needed and very welcome addition to help us treat our patients as best we can.” NICE deemed that, despite uncertainties in the cost-effectiveness evidence comparing evinacumab with lomitapide in adults with HoFH, overall there are cost savings with evinacumab compared with lomitapide. The NICE committee acknowledged that clinical trial evidence shows that Evkeeza, combined with other lipid-lowering therapies (LLTs), can lower LDL-C levels when statins and other LLTs have not reduced them enough. NICE also acknowledged that adolescent patients with HoFH on LLTs with or without lipoprotein apheresis have a high unmet need because LLTs have limited effectiveness in this patient population, lipoprotein apheresis can be traumatic and time consuming, and there is potential for an inequality of access if made available to adults and not adolescents. NICE considered that Evkeeza addresses an unmet need for new treatments to lower LDL-C, and that Evkeeza may improve adherence to treatment because it is administered once monthly (every 4 weeks) as an infusion. “We are delighted that patients with HoFH will have another tool to help manage their condition, especially as it is approved from age 12,” stated Jules Payne, CEO of Heart UK. "This recognition from NICE on the clinical and economic benefits of Evkeeza is a meaningful milestone for people living with HoFH in England and Wales,” said Erik Harris, chief commercial officer at Ultragenyx. “This highlights our commitment to working expeditiously on innovative ways to make Evkeeza broadly available to the HoFH community.” Evkeeza is now reimbursed and commercially available to prescribe for appropriate patients with HoFH in the U.S., England and Wales, Canada, Italy and Germany. 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 of premature atherosclerotic disease and cardiac events at an early age. About Evkeeza® (evinacumab) Evinacumab, the active substance in Evkeeza, binds 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 4 weeks. Evkeeza is approved by the Medicines and Healthcare products Regulatory Agency (MHRA) 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 homozygous familial hypercholesterolemia (HoFH). The effects of Evkeeza on cardiovascular morbidity and mortality have not been determined. 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 commercialization efforts for Evkeeza in HoFH in countries outside of the U.S. 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 Media Except 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 3, 2023, 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 MediaCarolyn Wang+1-415-225-5050media@ultragenyx.com

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) 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 5 to 11 years with homozygous familial hypercholesterolemia (HoFH). Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for children as young as 5 years old to control dangerously high levels of low-density lipoprotein cholesterol (LDL-C) caused by HoFH. Evkeeza initially received EC decision as an adjunct to diet and other lipid-lowering therapies in adolescents and adults aged 12 years and older with HoFH in June 2021. “This approval heralds a new era for treatment of children 5 to 11 years of age with HoFH,” stated Albert Wiegman, M.D., Ph.D. and Professor, Department of Paediatrics at Amsterdam University Medical Center. “The addition of Evkeeza to aggressive baseline lipid-lowering therapy nearly halves LDL-C levels in a disorder with an extremely high risk of premature cardiovascular disease in young patients, where achieving LDL-C goals otherwise isn’t possible. This EC decision follows the positive recommendation from the Committee for Medicinal Products for Human Use received in November 2023 and is based on the results of a Phase 3 open-label study in patients ages 5-11 years with HoFH.1 Despite treatment with other lipid-lowering therapies, children entered the Phase 3 trial with an average LDL-C level of 264 mg/dL, more than twice the target (<110 mg/dL) for paediatric patients with HoFH. With the addition of Evkeeza, children (n=14) were able to reduce their LDL-C by 48% at week 24 on average. Significant reductions were also observed in other key secondary endpoints including levels of apolipoprotein B (ApoB), non-high-density lipoprotein cholesterol (non-HDL-C) and total cholesterol. The safety profile of Evkeeza was consistent with the safety profile observed in adults and paediatric patients aged 12 years and older, with the additional adverse reaction of fatigue (reported in 3 patients). “It is with immense joy that the international community of parents and caregivers of children with HoFH welcomes this approval, because Evkeeza 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 to support their treatment goals.” Evkeeza is delivered via 60-minute intravenous infusion once monthly (every 4 weeks). The treatment is now reimbursed and commercially available to prescribe for appropriate patients with HoFH in the U.S., Canada, Italy and Germany. It is also available via early access schemes in Austria and France. "It is critical to reduce LDL levels as early as possible for all people living with HoFH especially given that children as young as 5 years of age are at risk for severe cardiac events that can be life threatening,”2 said Eric Crombez, M.D., chief medical officer at Ultragenyx. “With its novel mechanism, Evkeeza combined with other lipid-lowering therapies has demonstrated the ability to significantly reduce LDL cholesterol levels beyond historical standard of care, which could have a transformational impact for these younger patients.” The three-part, single-arm, open-label trial evaluated Evkeeza added to other lipid-lowering therapies in paediatric patients with HoFH aged 5 to 11 years. Part A (n=6) was a Phase 1b trial designed to assess the pharmacokinetics (PK), safety and tolerability of Evkeeza. Part B (n=14) evaluated the efficacy of Evkeeza during a 24-week treatment period and enrolled patients with an average age of 9 years. Among them, 86% were on statins, 93% were on ezetimibe, 50% were on LDL apheresis and 14% were on lomitapide. Patients received Evkeeza 15 mg/kg every four weeks delivered intravenously in addition to their baseline lipid-lowering treatment regimen. The primary endpoint was change in LDL-C at week 24. Secondary endpoints included the effect of Evkeeza on other lipid parameters (i.e., apolipoprotein B, non-high-density lipoprotein cholesterol, lipoprotein[a] and total cholesterol), efficacy by mutation status, safety and tolerability, immunogenicity and PK. Patients who completed Part A or B were allowed to, and all did, continue treatment in Part C (n=20), a completed Phase 3 extension trial. This trial was not designed to evaluate the effect of Evkeeza on cardiovascular events. Evkeeza was well tolerated in this patient population. The most common adverse events (AEs) occurring in >15% of patients were COVID-19 (n=15), pyrexia (n=5), headache (n=4), throat pain (oropharyngeal pain, n=4) as well as upper abdominal pain, diarrhea, vomiting, fatigue, nasopharyngitis, rhinitis and cough (all n=3). Most reported AEs were mild or moderate, and none led to study discontinuation. 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. 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 paediatric patients aged 5 years and older with homozygous familial hypercholesterolemia (HoFH). The effects of Evkeeza on cardiovascular morbidity and mortality have not been determined. 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 commercialization efforts for Evkeeza in countries outside of the U.S. The content above comes from the network. if any infringement, please contact us to modify.