FDA Accepts Agios’ Supplemental New Drug Application for PYRUKYND® (mitapivat) in Adult Patients with Non-Transfusion-Dependent and Transfusion-Dependent Alpha- or Beta-Thalassemia
Agios Pharmaceuticals (AGIO) announced the FDA's acceptance of their supplemental New Drug Application (sNDA) for PYRUKYND® (mitapivat) for treating adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The FDA assigned a Standard review classification with a PDUFA goal date of September 7, 2025.
The sNDA submission is supported by results from two Phase 3 trials: ENERGIZE and ENERGIZE-T, which evaluated mitapivat versus placebo in adults with non-transfusion-dependent and transfusion-dependent thalassemia. The ENERGIZE trial results were presented at the European Hematology Association 2024 Congress, while the ENERGIZE-T results were presented at the American Society of Hematology Annual Meeting in December 2024.
Agios Pharmaceuticals (AGIO) ha annunciato l'accettazione da parte della FDA della loro domanda supplementare di nuovo farmaco (sNDA) per PYRUKYND® (mitapivat) per il trattamento di pazienti adulti con talassemia alfa o beta, sia dipendenti che non dipendenti da trasfusioni. La FDA ha assegnato una classificazione di revisione standard con una data obiettivo PDUFA del 7 settembre 2025.
La presentazione della sNDA è supportata dai risultati di due studi di fase 3: ENERGIZE e ENERGIZE-T, che hanno valutato mitapivat rispetto al placebo in adulti con talassemia sia non dipendente che dipendente da trasfusioni. I risultati dello studio ENERGIZE sono stati presentati al Congresso dell'Associazione Europea di Ematologia 2024, mentre i risultati di ENERGIZE-T sono stati presentati alla Riunione Annuale dell'Associazione Americana di Ematologia nel dicembre 2024.
Agios Pharmaceuticals (AGIO) anunció la aceptación por parte de la FDA de su solicitud suplementaria de nuevo medicamento (sNDA) para PYRUKYND® (mitapivat) para el tratamiento de pacientes adultos con talasemia alfa o beta, tanto dependiente como no dependiente de transfusiones. La FDA ha asignado una clasificación de revisión estándar con una fecha límite PDUFA del 7 de septiembre de 2025.
La presentación de la sNDA está respaldada por los resultados de dos ensayos de fase 3: ENERGIZE y ENERGIZE-T, que evaluaron mitapivat frente a placebo en adultos con talasemia tanto dependiente como no dependiente de transfusiones. Los resultados del ensayo ENERGIZE se presentaron en el Congreso de la Asociación Europea de Hematología 2024, mientras que los resultados de ENERGIZE-T se presentaron en la Reunión Anual de la Sociedad Americana de Hematología en diciembre de 2024.
Agios Pharmaceuticals (AGIO)는 PYRUKYND® (mitapivat)의 추가 신약 신청서 (sNDA)가 비수혈 의존성과 수혈 의존성 알파 및 베타 탈라세미아 성인 환자 치료를 위한 FDA의 승인을 받았다고 발표했습니다. FDA는 2025년 9월 7일의 PDUFA 목표일과 함께 표준 검토 분류를 할당했습니다.
sNDA 제출은 비수혈 의존성 및 수혈 의존성 탈라세미아 성인에서 미타피바트와 플라세보를 비교한 두 가지 3상 시험인 ENERGIZE 및 ENERGIZE-T의 결과에 의해 뒷받침됩니다. ENERGIZE 시험 결과는 2024년 유럽 혈액학회에서 발표되었고, ENERGIZE-T 결과는 2024년 12월 미국 혈액학회 연례 회의에서 발표되었습니다.
Agios Pharmaceuticals (AGIO) a annoncé l'acceptation par la FDA de leur demande d'autorisation de mise sur le marché supplémentaire (sNDA) pour PYRUKYND® (mitapivat) pour le traitement des patients adultes atteints de thalassémie alpha ou bêta, dépendante ou non de transfusions. La FDA a attribué une classification de révision standard avec une date cible PDUFA du 7 septembre 2025.
La soumission du sNDA est soutenue par les résultats de deux essais de phase 3 : ENERGIZE et ENERGIZE-T, qui ont évalué le mitapivat par rapport à un placebo chez des adultes atteints de thalassémie, dépendants et non dépendants des transfusions. Les résultats de l'essai ENERGIZE ont été présentés au Congrès de l'Association Européenne d'Hématologie 2024, tandis que les résultats d'ENERGIZE-T ont été présentés lors de la Réunion Annuelle de la Société Américaine d'Hématologie en décembre 2024.
Agios Pharmaceuticals (AGIO) gab bekannt, dass die FDA ihren Antrag auf ein zusätzliches neues Medikament (sNDA) für PYRUKYND® (mitapivat) zur Behandlung von erwachsenen Patienten mit nicht transfusionsabhängiger und transfusionsabhängiger Alpha- oder Beta-Thalassämie akzeptiert hat. Die FDA hat eine Standardprüfungszuordnung mit einem PDUFA-Zieldatum vom 7. September 2025 vergeben.
Die Einreichung des sNDA wird durch die Ergebnisse von zwei Phase-3-Studien unterstützt: ENERGIZE und ENERGIZE-T, die mitapivat gegen Placebo bei Erwachsenen mit nicht transfusionsabhängiger und transfusionsabhängiger Thalassämie bewerteten. Die Ergebnisse der ENERGIZE-Studie wurden beim Kongress der Europäischen Hämatologiegesellschaft 2024 präsentiert, während die Ergebnisse von ENERGIZE-T auf dem Jahresmeeting der Amerikanischen Hämatologischen Gesellschaft im Dezember 2024 vorgestellt wurden.
- FDA acceptance of sNDA for PYRUKYND® expansion into thalassemia treatment
- Clear PDUFA date set for September 7, 2025
- Potential market expansion into both transfusion-dependent and non-transfusion-dependent patients
- Standard review classification rather than priority review
- Long review timeline extending to September 2025
Insights
The FDA's acceptance of Agios' sNDA for PYRUKYND® represents a important milestone in addressing the significant unmet medical need in thalassemia treatment. The September 7, 2025 PDUFA date sets a clear timeline for potential market entry. The drug's unique mechanism as a pyruvate kinase activator could revolutionize thalassemia treatment by offering an oral option for both transfusion-dependent and non-dependent patients.
The dual Phase 3 trial approach (ENERGIZE and ENERGIZE-T) demonstrates robust clinical development strategy, targeting distinct patient populations. Notably, the inclusion of both alpha- and beta-thalassemia patients broadens the potential market significantly. With
Think of PYRUKYND® as a master key - it's designed to work across different types of thalassemia, regardless of whether patients need regular blood transfusions or not. This versatility could make it a go-to treatment option, potentially reducing healthcare costs associated with current transfusion-dependent care.
This FDA acceptance marks a significant catalyst for AGIO's market position. PYRUKYND® is already approved for pyruvate kinase deficiency, generating
The Standard review classification, while not as fast as Priority Review, suggests a well-established safety profile. Market penetration could be swift post-approval due to competition in the oral treatment space for thalassemia. Current treatment costs for transfusion-dependent patients can exceed
For everyday investors: Imagine PYRUKYND® as adding a new floor to an existing building - it's expanding the drug's reach into a bigger market, which could significantly boost Agios' revenue potential. The stock might see increased attention as we approach the September PDUFA date, particularly if interim updates remain positive.
CAMBRIDGE, Mass., Jan. 08, 2025 (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 U.S. Food and Drug Administration (FDA) accepted the company’s supplemental New Drug Application (sNDA) for PYRUKYND® (mitapivat) for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The review classification for this application is Standard and the Prescription Drug User Fee Act (PDUFA) goal date is September 7, 2025.
“Thalassemia is a rare, lifelong inherited blood disorder that causes chronic anemia and can lead to severe complications, including organ damage, stroke, and other serious health issues, with patients today having limited or no effective treatment options,” said Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D at Agios. “We look forward to collaborating with the FDA in the coming months as they continue to review our application, with the goal of bringing PYRUKYND, a disease-modifying oral medication, to thalassemia patients regardless of their genotype or transfusion needs.”
The sNDA is based on the results from the ENERGIZE and ENERGIZE-T Phase 3 trials evaluating mitapivat versus placebo in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, respectively. The ENERGIZE randomized clinical trial results were presented at the European Hematology Association 2024 Hybrid Congress in June 2024, and the ENERGIZE-T randomized clinical trial results were presented at the 66ᵗʰ American Society of Hematology Annual Meeting and Exposition in December 2024.
About PYRUKYND® (mitapivat)
U.S. INDICATION
PYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.
U.S. 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.
Hepatocellular Injury in Another Condition: In patients with another condition treated with PYRUKYND at a higher dose than that recommended for patients with PK deficiency, liver injury has been observed. These events were characterized by a time to onset within the first 6 months of treatment with peak elevations of alanine aminotransferase of >5× upper limit of normal (ULN) with or without jaundice. All patients discontinued treatment with PYRUKYND, and these events improved upon treatment discontinuation.
Obtain liver tests prior to the initiation of PYRUKYND and monthly thereafter for the first 6 months and as clinically indicated. Interrupt PYRUKYND if clinically significant increases in liver tests are observed or alanine aminotransferase is >5x ULN. Discontinue PYRUKYND if hepatic injury due to PYRUKYND is suspected.
Adverse Reactions: The most common adverse reactions including laboratory abnormalities (≥
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 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’ expectations for the FDA’s review of its sNDA for PYRUKYND® in alpha-and-beta thalassemia; 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 is the PDUFA date for AGIO's PYRUKYND® thalassemia application?
What type of FDA review classification did AGIO's PYRUKYND® receive for thalassemia?
Which Phase 3 trials support AGIO's PYRUKYND® sNDA for thalassemia?
What patient populations will AGIO's PYRUKYND® target in thalassemia?
