Agios Announces FDA Orphan Drug Designation Granted to Tebapivat (AG-946) for Treatment of Myelodysplastic Syndromes (MDS)
Agios Pharmaceuticals (Nasdaq: AGIO) has received FDA orphan drug designation for its novel pyruvate kinase activator tebapivat (AG-946) in the treatment of myelodysplastic syndromes (MDS). This designation supports the development of medicines for rare disorders affecting fewer than 200,000 people in the U.S. and offers incentives such as tax credits and potential market exclusivity.
Tebapivat aims to be the first oral therapy addressing anemia due to ineffective erythropoiesis in lower-risk MDS, which affects 75,000-80,000 patients in the U.S. and EU5. Agios has completed a Phase 2a study and is initiating a Phase 2b study of tebapivat in lower-risk MDS. The company's lead PK activator, mitapivat, has previously received FDA orphan drug designation for other rare blood disorders.
Agios Pharmaceuticals (Nasdaq: AGIO) ha ricevuto la designazione di farmaco orfano FDA per il suo nuovo attivatore della piruvato chinasi tebapivat (AG-946) nel trattamento della sindrome mielodisplastica (MDS). Questa designazione supporta lo sviluppo di medicinali per malattie rare che colpiscono meno di 200.000 persone negli Stati Uniti e offre incentivi come crediti d'imposta e potenziale esclusiva di mercato.
Tebapivat punta a essere la prima terapia orale per affrontare l'anemia dovuta a eritropoiesi inefficace nei MDS a basso rischio, che colpiscono 75.000-80.000 pazienti negli Stati Uniti e nei 5 principali paesi dell'UE. Agios ha completato uno studio di Fase 2a e sta avviando uno studio di Fase 2b di tebapivat nei MDS a basso rischio. L'attivatore PK principale dell'azienda, mitapivat, ha precedentemente ricevuto la designazione di farmaco orfano FDA per altre malattie ematologiche rare.
Agios Pharmaceuticals (Nasdaq: AGIO) ha recibido la designación de medicamento huérfano por parte de la FDA para su nuevo activador de la quinasa de piruvato tebapivat (AG-946) en el tratamiento de síndromes mielodisplásicos (MDS). Esta designación respalda el desarrollo de medicamentos para trastornos raros que afectan a menos de 200,000 personas en EE.UU. y ofrece incentivos como créditos fiscales y potencial exclusividad de mercado.
Tebapivat tiene como objetivo ser la primera terapia oral que aborde la anemia debida a eritropoyesis ineficaz en MDS de bajo riesgo, que afecta a 75,000-80,000 pacientes en EE.UU. y EU5. Agios ha completado un estudio de Fase 2a y está iniciando un estudio de Fase 2b de tebapivat en MDS de bajo riesgo. El principal activador de PK de la compañía, mitapivat, ya ha recibido la designación de medicamento huérfano de la FDA para otros trastornos sanguíneos raros.
Agios Pharmaceuticals (Nasdaq: AGIO)는 FDA의 희귀약품 지정을 신규 피루브산 키나제 활성제 테바피바트 (AG-946)에 대해 골수형성이상증후군 (MDS) 치료에 받아들였습니다. 이 지정은 미국에서 20만 명 미만의 사람에게 영향을 미치는 희귀 질환을 위한 의약품 개발을 지원하며, 세금 공제 및 시장 독점의 잠재적 혜택과 같은 인센티브를 제공합니다.
테바피바트는 낮은 위험 MDS에서의 비효율적 적혈구 생성으로 인한 빈혈을 다루는 첫 번째 경구 요법이 되는 것을 목표로 하며, 이는 미국과 EU5에서 75,000-80,000 명의 환자에게 영향을 미칩니다. Agios는 2a상 연구를 완료하고 낮은 위험 MDS에서 테바피바트에 대한 2b상 연구를 시작하고 있습니다. 회사의 주요 PK 활성제인 미타피바트는 이전에 다른 희귀 혈액 질환에 대해 FDA의 희귀약품 지명을 받았습니다.
Agios Pharmaceuticals (Nasdaq: AGIO) a reçu la désignation de médicament orphelin de la FDA pour son nouvel activateur de la pyruvate kinase tebapivat (AG-946) dans le traitement des syndromes myélodysplasiques (MDS). Cette désignation soutient le développement de médicaments pour des troubles rares touchant moins de 200 000 personnes aux États-Unis et offre des incitations telles que des crédits d'impôt et une éventuelle exclusivité sur le marché.
Tebapivat vise à être la première thérapie orale visant l'anémie due à une érythropoïèse inefficace dans les MDS à faible risque, qui affecte 75 000 à 80 000 patients aux États-Unis et dans l'UE5. Agios a terminé une étude de Phase 2a et initie une étude de Phase 2b de tebapivat dans les MDS à faible risque. Le principal activateur PK de l'entreprise, mitapivat, a déjà reçu la désignation de médicament orphelin de la FDA pour d'autres troubles sanguins rares.
Agios Pharmaceuticals (Nasdaq: AGIO) hat von der FDA die Auszeichnung als Orphan Drug für seinen neuartigen Pyruvatkinase-Aktivator tebapivat (AG-946) zur Behandlung von myelodysplastischen Syndromen (MDS) erhalten. Diese Auszeichnung unterstützt die Entwicklung von Arzneimitteln für seltene Erkrankungen, die weniger als 200.000 Menschen in den USA betreffen, und bietet Anreize wie Steuergutschriften und potenzielle Marktexklusivität.
Tebapivat zielt darauf ab, die erste orale Therapie zu sein, die Anämie aufgrund ineffizienter Erythropoese bei MDS mit niedrigem Risiko behandelt, was 75.000-80.000 Patienten in den USA und EU5 betrifft. Agios hat eine Phase-2a-Studie abgeschlossen und startet eine Phase-2b-Studie zu tebapivat bei MDS mit niedrigem Risiko. Der führende PK-Aktivator des Unternehmens, mitapivat, erhielt bereits die Orphan-Drug-Auszeichnung der FDA für andere seltene Bluterkrankungen.
- FDA granted orphan drug designation for tebapivat in MDS treatment
- Potential for tax credits and 7 years of market exclusivity
- Tebapivat aims to be the first oral therapy for anemia in lower-risk MDS
- Completed Phase 2a study and initiating Phase 2b study for tebapivat in lower-risk MDS
- Large target market of 75,000-80,000 patients in the U.S. and EU5
- Tebapivat is not yet approved for use by any regulatory authority
- Still in clinical trial phase, with no guarantee of success or approval
Insights
The FDA's orphan drug designation for tebapivat in MDS is a significant milestone for Agios Pharmaceuticals. This designation highlights the unmet medical need in lower-risk MDS, particularly in addressing anemia due to ineffective erythropoiesis. With
The completion of the Phase 2a study and initiation of a Phase 2b study indicate promising progress in tebapivat's development. If successful, it could become the first oral therapy for this specific aspect of MDS, potentially improving quality of life for patients who currently lack effective treatment options. The orphan drug designation also provides Agios with financial incentives and the possibility of seven years of market exclusivity, which could significantly boost the drug's commercial potential upon approval.
The orphan drug designation for tebapivat is a positive development for Agios Pharmaceuticals. This designation offers several financial benefits, including tax credits and exemptions from certain FDA fees, which could significantly reduce the costs associated with clinical trials and drug development. The potential for seven years of market exclusivity is particularly valuable, as it could provide Agios with a substantial competitive advantage and protect future revenue streams.
Investors should note that while this news is encouraging, tebapivat is still in early-stage development. The initiation of the Phase 2b study suggests continued investment in R&D, which may impact short-term financials. However, if successful, tebapivat could address a sizeable market of 75,000-80,000 patients, potentially translating to significant future revenue. This development also demonstrates Agios's growing pipeline and expertise in PK activation therapies, which could enhance the company's long-term value proposition.
CAMBRIDGE, Mass., Sept. 11, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company’s novel pyruvate kinase (PK) activator tebapivat (AG-946) for the treatment of myelodysplastic syndromes (MDS).
“Receiving orphan drug designation for tebapivat in MDS underscores the importance of bringing new oral treatment options to patients suffering from this rare disease,” said Sarah Gheuens, M.D., Ph.D., chief medical officer and head R&D at Agios. “We aim to deliver the first oral therapy that addresses anemia due to ineffective erythropoiesis in lower-risk MDS, which affects approximately 75,000-80,000 patients in the U.S. and EU5 and accounts for approximately
Agios completed a Phase 2a study of tebapivat in lower-risk MDS late last year and is currently initiating a Phase 2b study of tebapivat in lower-risk MDS.
The FDA’s Office of Orphan Drug Products grants orphan drug designation to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S. Under the Orphan Drug Act, orphan drug designation qualifies a company for incentives, including tax credits, exemptions from certain FDA fees for clinical trials, and the potential for seven years of market exclusivity following drug approval.
Mitapivat, the company’s lead PK activator, was previously granted FDA orphan drug designation for the treatment of PK deficiency, thalassemia, and sickle cell disease.
Tebapivat is not approved for use by any regulatory authority.
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, 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), tebapivat (AG-946), TMPRSS6 siRNA and AG-181, Agios’ PAH stabilizer; Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including PYRUKYND®, tebapivat and AG-181; Agios’ use of proceeds from the transaction with Royalty Pharma; potential U.S. net sales of vorasidenib and potential future royalty payments; Agios’ strategic vision and goals, including its key milestones for 2024; 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 milestone or royalty payments related to the sale of its oncology business or 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
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1AB
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FAQ
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