European Commission (EC) Extends the Approval of Evkeeza® (evinacumab) to Children as Young as 6-months Old with Homozygous Familial Hypercholesterolemia (HoFH)
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that the European Commission has extended the approval of Evkeeza® (evinacumab) to treat children aged 6-months and older with homozygous familial hypercholesterolemia (HoFH). Evkeeza is the first medicine in the EU approved for children as young as 6-months old with this ultrarare inherited condition that causes dangerously high cholesterol levels.
The approval is based on model-based extrapolation analysis predicting similar or higher LDL-C reduction in young patients compared to adults when receiving a 15 mg/kg dose every 4 weeks. Supportive data from five patients aged 1-4 years showed clinically meaningful LDL-C reduction consistent with older patients. The safety profile is expected to be similar to that in older pediatric patients.
Evkeeza is currently reimbursed and commercially available in eight countries including the UK, U.S., Canada, and Japan, with early access schemes in 13 additional countries.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) ha annunciato che la Commissione Europea ha esteso l'approvazione di Evkeeza® (evinacumab) per il trattamento di bambini di età pari o superiore a 6 mesi affetti da ipercolesterolemia familiare omozigote (HoFH). Evkeeza è il primo medicinale nell'UE approvato per bambini di appena 6 mesi con questa condizione ultrarara ereditaria che causa pericolosamente alti livelli di colesterolo.
L'approvazione si basa su un'analisi di estrapolazione basata su modelli che prevede una riduzione simile o maggiore del LDL-C nei pazienti giovani rispetto agli adulti quando si riceve una dose di 15 mg/kg ogni 4 settimane. Dati di supporto provenienti da cinque pazienti di età compresa tra 1 e 4 anni hanno mostrato una riduzione clinicamente significativa del LDL-C, coerente con quella osservata in pazienti più anziani. Il profilo di sicurezza è atteso essere simile a quello dei pazienti pediatrici più grandi.
Evkeeza è attualmente rimborsato ed è commercialmente disponibile in otto paesi, tra cui Regno Unito, Stati Uniti, Canada e Giappone, con schemi di accesso anticipato in ulteriori 13 paesi.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) anunció que la Comisión Europea ha ampliado la aprobación de Evkeeza® (evinacumab) para tratar a niños de 6 meses de edad y mayores con hipercolesterolemia familiar homocigota (HoFH). Evkeeza es el primer medicamento en la UE aprobado para niños tan jóvenes como los de 6 meses con esta condición ultrarrara hereditaria que causa niveles de colesterol peligrosamente altos.
La aprobación se basa en un análisis de extrapolación basado en modelos que predice una reducción del LDL-C similar o mayor en pacientes jóvenes en comparación con adultos al recibir una dosis de 15 mg/kg cada 4 semanas. Los datos de respaldo de cinco pacientes de 1 a 4 años mostraron una reducción clínica significativa del LDL-C consistente con la de pacientes mayores. Se espera que el perfil de seguridad sea similar al de los pacientes pediátricos mayores.
Evkeeza actualmente está reembolsado y disponible comercialmente en ocho países, incluidos el Reino Unido, EE. UU., Canadá y Japón, con esquemas de acceso anticipado en 13 países adicionales.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE)는 유럽연합 집행위원회가 Evkeeza® (evinacumab)의 승인을 연장하여 6개월 이상의 어린이에게 동형접합 가족성 고콜레스테롤혈증 (HoFH)을 치료할 수 있게 되었다고 발표했습니다. Evkeeza는 이 초희귀 유전 질환으로 인해 위험한 고지혈증을 앓고 있는 6개월 된 아동에게 유럽에서 승인된 최초의 약물입니다.
이번 승인은 모델 기반의 추정 분석에 기반하여, 4주마다 15 mg/kg의 용량을 투여받는 젊은 환자들에서 성인에 비해 유사하거나 더 높은 LDL-C 감소가 예측된 결과입니다. 1세부터 4세까지의 5명의 환자로부터 얻은 지원 데이터는 남은 환자들과 일치하는 임상적으로 의미 있는 LDL-C 감소를 보여주었습니다. 안전성 프로파일은 더 큰 어린이 환자의 것과 유사할 것으로 예상됩니다.
Evkeeza는 현재 영국, 미국, 캐나다 및 일본을 포함한 8개국에서 보험 적용을 받으며 상업적으로 이용 가능하며, 추가 13개국에서는 조기 접근 프로그램이 운영되고 있습니다.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) a annoncé que la Commission Européenne a étendu l'approbation de Evkeeza® (evinacumab) pour traiter les enfants âgés de 6 mois et plus atteints d'hypercholestérolémie familiale homozygote (HoFH). Evkeeza est le premier médicament dans l'UE approuvé pour des enfants aussi jeunes que 6 mois souffrant de cette condition génétique ultra-rare qui entraîne des niveaux de cholestérol dangereusement élevés.
L'approbation est basée sur une analyse d'extrapolation par modélisation prédisant une réduction similaire ou supérieure du LDL-C chez les jeunes patients par rapport aux adultes lors de l'administration d'une dose de 15 mg/kg toutes les 4 semaines. Les données soutenant cinq patients âgés de 1 à 4 ans ont montré une réduction cliniquement significative du LDL-C, conforme à celle observée chez des patients plus âgés. Le profil de sécurité devrait être similaire à celui observé chez les jeunes patients.
Evkeeza est actuellement remboursé et commercialisé dans huit pays, y compris le Royaume-Uni, les États-Unis, le Canada et le Japon, avec des programmes d'accès précoce dans 13 pays supplémentaires.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) kündigte an, dass die Europäische Kommission die Zulassung für Evkeeza® (evinacumab) zur Behandlung von Kindern ab 6 Monaten mit homozygoter familiärer Hypercholesterinämie (HoFH) erweitert hat. Evkeeza ist das erste Medikament in der EU, das für Kinder ab 6 Monate mit dieser ultrararen erblichen Erkrankung, die gefährlich hohe Cholesterinwerte verursacht, zugelassen ist.
Die Zulassung basiert auf einer modellbasierten Extrapolationsanalyse, die eine ähnliche oder höhere LDL-C-Reduktion bei jungen Patienten im Vergleich zu Erwachsenen bei einer Dosis von 15 mg/kg alle 4 Wochen vorhersagt. Unterstützende Daten von fünf Patienten im Alter von 1 bis 4 Jahren zeigten eine klinisch signifikante LDL-C-Reduktion, die mit älteren Patienten übereinstimmte. Das Sicherheitsprofil wird voraussichtlich ähnlich sein wie das bei älteren pädiatrischen Patienten.
Evkeeza wird derzeit in acht Ländern, darunter das Vereinigte Königreich, die USA, Kanada und Japan, erstattet und kommerziell angeboten, mit frühen Zugangsprogrammen in weiteren 13 Ländern.
- First EU-approved treatment for HoFH patients aged 6-months to 5 years
- Demonstrated efficacy with significant LDL-C reduction in young patients
- Already commercially available in 8 major markets
- Available via early access in 13 additional countries
- None.
Insights
The EC approval extension for Evkeeza® to treat HoFH in children as young as 6 months represents a significant therapeutic advancement. The efficacy data showing comparable or superior LDL-C reduction in young children versus adults is particularly compelling. Model-based extrapolation analysis combined with real-world compassionate use data in five patients aged 1-4 years demonstrates clinically meaningful LDL-C reductions.
The expansion into the 6-months to 5-years age group addresses a critical unmet need in HoFH treatment. Early intervention is important as these patients face severe cardiovascular risks from dangerously elevated LDL-C levels. The consistent safety profile across age groups and absence of new safety concerns in compassionate use strengthens the risk-benefit profile.
For investors, this approval expands Evkeeza's addressable market in the EU, though the ultra-rare nature of HoFH means the direct revenue impact may be modest. The broader significance lies in validating Ultragenyx's capability in rare disease drug development and commercialization.
The commercial implications of this approval are nuanced. While HoFH affects an extremely small patient population - approximately 1 in 300,000 people - rare disease therapies typically command premium pricing. The approval in children as young as 6 months strengthens Evkeeza's market position as the first and only approved therapy for this age group in the EU.
Current reimbursement and commercial availability in 8 key markets (UK, US, Canada, Italy, Japan, Netherlands, Spain, Luxembourg) plus early access in 13 additional countries indicates strong market penetration strategy. This geographic footprint, combined with the expanded age indication, positions Ultragenyx to capture a larger share of the HoFH market.
The lack of treatment alternatives for very young HoFH patients suggests potential for rapid adoption where reimbursement is secured. The unmet medical need and first-mover advantage in this age group could support favorable pricing and reimbursement negotiations.
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 cholesterol
BASEL, 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 (>
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 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 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-/).
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FAQ
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