European Commission Adopts Positive Decision for Orphan Medicinal Product Designation of Agios’ Mitapivat in Sickle Cell Disease
Agios Pharmaceuticals (AGIO) has received orphan medicinal product designation from the European Commission for mitapivat, their oral PK activator, for treating sickle cell disease. This follows the FDA's orphan drug designation granted in November 2020. The European designation provides benefits including reduced fees and 10-year market exclusivity for conditions affecting fewer than 5 in 10,000 EU individuals.
The company's Phase 3 RISE UP study evaluating mitapivat's efficacy and safety in sickle cell disease is fully enrolled, with results expected in late 2025. This designation highlights the urgent need for new sickle cell disease treatments and mitapivat's potential to provide meaningful benefits to patients.
Agios Pharmaceuticals (AGIO) ha ricevuto la designazione di farmaco orfano dalla Commissione Europea per mitapivat, il loro attivatore PK orale, per il trattamento della malattia falciforme. Questo segue la designazione di farmaco orfano concessa dalla FDA nel novembre 2020. La designazione europea offre vantaggi tra cui riduzione delle tasse e un'esclusività di mercato di 10 anni per le condizioni che colpiscono meno di 5 persone su 10.000 nell'UE.
Lo studio di Fase 3 RISE UP dell'azienda, che valuta l'efficacia e la sicurezza di mitapivat nella malattia falciforme, è completamente arruolato, con risultati attesi per la fine del 2025. Questa designazione evidenzia l'urgenza di nuovi trattamenti per la malattia falciforme e il potenziale di mitapivat di fornire benefici significativi ai pazienti.
Agios Pharmaceuticals (AGIO) ha recibido la designación de medicamento huérfano de la Comisión Europea para mitapivat, su activador PK oral, para el tratamiento de la enfermedad de células falciformes. Esto sigue a la designación de medicamento huérfano otorgada por la FDA en noviembre de 2020. La designación europea ofrece beneficios como tarifas reducidas y 10 años de exclusividad de mercado para condiciones que afectan a menos de 5 de cada 10,000 personas en la UE.
El estudio de Fase 3 RISE UP de la empresa, que evalúa la eficacia y seguridad de mitapivat en la enfermedad de células falciformes, está completamente inscrito, con resultados esperados para finales de 2025. Esta designación destaca la urgente necesidad de nuevos tratamientos para la enfermedad de células falciformes y el potencial de mitapivat de proporcionar beneficios significativos a los pacientes.
Agios Pharmaceuticals (AGIO)는 유럽 위원회로부터 mitapivat에 대해 희귀 의약품 지정을 받았습니다. 이 약은 겸상 적혈구 질환 치료를 위한 경구용 PK 활성제입니다. 이는 2020년 11월 FDA에서 받은 희귀 의약품 지정을 뒤따르는 것입니다. 유럽의 이 지명은 10,000명 중 5명 이하의 건강 문제에 대한 혜택으로 수수료 감소 및 10년의 시장 독점권을 포함합니다.
회사의 3상 RISE UP 연구는 겸상 적혈구 질환에서 mitapivat의 효능과 안전성을 평가하고 있으며, 완전히 등록되었습니다. 결과는 2025년 말에 예상됩니다. 이번 지명은 새로운 겸상 적혈구 질환 치료의 절박한 필요성을 강조하며, mitapivat가 환자에게 의미 있는 혜택을 제공할 수 있는 잠재력을 가지고 있음을 시사합니다.
Agios Pharmaceuticals (AGIO) a reçu la désignation de médicament orphelin de la Commission Européenne pour mitapivat, leur activateur PK oral, pour le traitement de la drépanocytose. Cela fait suite à la désignation de médicament orphelin accordée par la FDA en novembre 2020. La désignation européenne offre des avantages tels que des frais réduits et une exclusivité de marché de 10 ans pour les pathologies touchant moins de 5 personnes sur 10 000 dans l'UE.
L'étude de Phase 3 RISE UP de l'entreprise, qui évalue l'efficacité et la sécurité de mitapivat dans la drépanocytose, est complètement recrutée, avec des résultats attendus fin 2025. Cette désignation souligne le besoin urgent de nouveaux traitements contre la drépanocytose et le potentiel de mitapivat à offrir des bénéfices significatifs aux patients.
Agios Pharmaceuticals (AGIO) hat von der Europäischen Kommission die Orphan-Medicinal-Product-Auszeichnung für mitapivat, ihren oralen PK-Aktivator zur Behandlung der Sichelzellenkrankheit, erhalten. Dies folgt der Orphan-Drug-Auszeichnung der FDA, die im November 2020 gewährt wurde. Die europäische Auszeichnung bietet Vorteile wie reduzierte Gebühren und eine 10-jährige Marktexklusivität für Bedingungen, die weniger als 5 von 10.000 Personen in der EU betreffen.
Die Phase-3-Studie RISE UP des Unternehmens, die die Wirksamkeit und Sicherheit von mitapivat bei Sichelzellenkrankheit bewertet, ist vollständig eingeschrieben, und die Ergebnisse werden für Ende 2025 erwartet. Diese Auszeichnung hebt den dringenden Bedarf an neuen Behandlungen für die Sichelzellenkrankheit hervor und zeigt das Potenzial von mitapivat, um bedeutende Vorteile für die Patienten zu bieten.
- Received European Commission orphan medicinal product designation for mitapivat
- Secured 10-year market exclusivity in the EU
- Phase 3 RISE UP study fully enrolled
- Previously received FDA orphan drug designation
- Results from Phase 3 RISE UP study not available until late 2025
Insights
The orphan drug designation from the European Commission represents a significant regulatory milestone for Agios's mitapivat in sickle cell disease. This designation, coupled with the existing FDA orphan drug status, provides substantial benefits including 10 years of market exclusivity in the EU and reduced regulatory fees. The fully enrolled Phase 3 RISE UP study, with results expected in late 2025, will be important in determining mitapivat's clinical efficacy.
Sickle cell disease represents an underserved market with significant unmet medical needs. The orphan designation validates mitapivat's potential as a novel oral treatment option, particularly noteworthy given the current treatment landscape which largely relies on more invasive therapies. The development of an oral small molecule treatment could significantly improve patient compliance and quality of life if proven effective in Phase 3 trials.
The European Commission's orphan designation strengthens Agios's market position and potential revenue streams. The 10-year market exclusivity period is particularly valuable, providing a significant competitive advantage and protection for future revenues in the EU market. For a company with a
This regulatory milestone reduces development costs through fee reductions and potentially accelerated approval pathways. While revenue impact is still dependent on Phase 3 results and eventual commercialization, the orphan designation significantly de-risks the European market entry strategy. The sickle cell disease market represents a substantial commercial opportunity, with the orphan status helping to maximize the potential return on R&D investment.
CAMBRIDGE, Mass., Dec. 18, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today announced that the European Commission has adopted a positive decision for the designation of mitapivat, an oral, small molecule PK activator, as an orphan medicinal product (OMP) for the treatment of sickle cell disease. Earlier, in November 2020, the U.S. Food and Drug Administration (FDA) also granted orphan drug designation to mitapivat for sickle cell disease.
“Alongside the FDA’s orphan drug designation in the U.S., the European Commission’s orphan medicinal product designation for mitapivat underscores the urgent need for novel therapies for sickle cell disease and highlights its potential to provide clinically meaningful benefits to patients navigating this debilitating condition,” said Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D at Agios. “With the trial fully enrolled, we look forward to sharing the results of our Phase 3 RISE UP study evaluating the efficacy and safety of mitapivat in sickle cell disease with the community in late 2025.”
The European Commission offers OMP designation to innovative therapies that address life-threatening or chronically debilitating conditions affecting fewer than five in 10,000 individuals in the European Union, and that have the potential to provide a significant benefit over existing treatments. This designation provides extensive benefits to encourage the development of these medicines, including reduced fees and a 10-year period of market exclusivity.
About Phase 2/3 RISE UP Study
The RISE UP Phase 2 and 3 studies are evaluating the efficacy and safety of mitapivat in sickle cell disease patients who are 16 years of age or older, have had between two and 10 sickle cell pain crises in the past 12 months, and have hemoglobin within the range of 5.5 to 10.5 g/dL during screening. The Phase 2 and Phase 3 studies are conducted under a single operationally seamless Phase 2/3 protocol. The two studies enrolled different participants and achieved operational efficiency through leveraging the same sites, vendors and other resources.
The Phase 2 study included a 12-week randomized, placebo-controlled period in which participants were randomized in a 1:1:1 ratio to receive 50 mg mitapivat twice daily, 100 mg mitapivat twice daily or matched placebo. The primary endpoints were hemoglobin response, defined as ≥1.0 g/dL increase in average hemoglobin concentration from Week 10 through Week 12 compared to baseline, and safety. In December 2023, Agios presented positive results from the Phase 2 study at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition.
The Phase 3 study includes a 52-week randomized, placebo-controlled period in which participants will be randomized in a 2:1 ratio to receive 100 mg of mitapivat twice daily or matched placebo. The primary endpoints are hemoglobin response, defined as a ≥1.0 g/dL increase in average hemoglobin concentration from Week 24 through Week 52 compared with baseline, and annualized rate of sickle cell pain crises. In October 2024, Agios announced that enrollment in the Phase 3 study had been completed, with more than 200 patients enrolled worldwide.
Participants who complete the double-blind period of the Phase 2 or Phase 3 studies will have the option to move into a 216-week open-label extension period to receive mitapivat.
About PYRUKYND® (mitapivat)
PYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency in the United States, and for the treatment of PK deficiency in adult patients in the European Union.
IMPORTANT SAFETY INFORMATION
Acute Hemolysis: Acute hemolysis with subsequent anemia has been observed following abrupt interruption or discontinuation of PYRUKYND in a dose-ranging study. Avoid abruptly discontinuing PYRUKYND. Gradually taper the dose of PYRUKYND to discontinue treatment if possible. When discontinuing treatment, monitor patients for signs of acute hemolysis and anemia including jaundice, scleral icterus, dark urine, dizziness, confusion, fatigue, or shortness of breath.
Adverse Reactions: Serious adverse reactions occurred in
Drug Interactions:
- Strong CYP3A Inhibitors and Inducers: Avoid concomitant use.
- Moderate CYP3A Inhibitors: Do not titrate PYRUKYND beyond 20 mg twice daily.
- Moderate CYP3A Inducers: Consider alternatives that are not moderate inducers. If there are no alternatives, adjust PYRUKYND dosage.
- Sensitive CYP3A, CYP2B6, CYP2C Substrates Including Hormonal Contraceptives: Avoid concomitant use with substrates that have narrow therapeutic index.
- UGT1A1 Substrates: Avoid concomitant use with substrates that have narrow therapeutic index.
- P-gp Substrates: Avoid concomitant use with substrates that have narrow therapeutic index.
Hepatic Impairment: Avoid use of PYRUKYND in patients with moderate and severe hepatic impairment.
Please see full Prescribing Information and Summary of Product Characteristics for PYRUKYND.
About Agios
Agios is the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with rare diseases. In the U.S., Agios markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. Building on the company's deep scientific expertise in classical hematology and leadership in the field of cellular metabolism and rare hematologic diseases, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, myelodysplastic syndrome (MDS)-associated anemia and phenylketonuria (PKU). In addition to its clinical pipeline, Agios is advancing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera. For more information, please visit the company’s website at www.agios.com.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of PYRUKYND® (mitapivat); Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including PYRUKYND®; Agios’ strategic vision and goals, including its key milestones; and the potential benefits of Agios’ strategic plans and focus. The words “anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,” “will,” “vision,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios’ current expectations and beliefs. For example, there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios’ product candidates will successfully continue. There can be no guarantee that any positive developments in Agios’ business will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation: risks and uncertainties related to the impact of pandemics or other public health emergencies to Agios’ business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Agios’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios’ ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Agios' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios’ ability to establish and maintain key collaborations; uncertainty regarding any royalty payments related to the sale of its oncology business or any milestone or royalty payments related to its in-licensing of TMPRSS6 siRNA, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of Agios’ cash and cash equivalents; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in Agios’ public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
Contacts:
Investor Contact
Chris Taylor, VP, Investor Relations and Corporate Communications
Agios Pharmaceuticals
IR@agios.com
Media Contact
Eamonn Nolan, Senior Director, Corporate Communications
Agios Pharmaceuticals
Media@agios.com
FAQ
When will Agios (AGIO) release Phase 3 RISE UP study results for mitapivat in sickle cell disease?
What benefits does the EU orphan medicinal product designation provide AGIO's mitapivat?
When did AGIO receive FDA orphan drug designation for mitapivat in sickle cell disease?
What is the prevalence requirement for EU orphan medicinal product designation that AGIO's mitapivat met?
