Agios Pharmaceuticals Receives FDA Orphan Drug Designation for Tebapivat in MDS Treatment
Agios Pharmaceuticals has achieved a significant milestone with the announcement that tebapivat (AG-946), its innovative treatment for myelodysplastic syndromes (MDS), has been granted orphan drug designation (ODD) by the US Food and Drug Administration (FDA). This pivotal development follows the successful completion of a Phase IIa trial for tebapivat and the commencement of a Phase IIb study, targeting the same indication.
The FDA’s Office of Orphan Drug Products assigns ODD to support the progress of medical solutions for rare conditions, defined as those affecting fewer than 200,000 people in the United States. This designation is crucial for Agios Pharmaceuticals as it comes with a host of advantages, including tax credits, exemptions from certain FDA fees, and the potential for seven years of market exclusivity upon the drug’s approval.
Expressing optimism about the designation, Agios’ research and development chief medical officer, Sarah Gheuens, highlighted the significance of tebapivat in addressing MDS, a rare and debilitating disease. “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 Gheuens. She further added, “We aim to deliver the first oral therapy that addresses anaemia due to ineffective erythropoiesis in lower-risk MDS, which affects approximately 75,000 to 80,000 patients in France, Germany, Italy, Spain, the UK, and the US, and accounts for approximately 70% of MDS cases.”
Agios Pharmaceuticals is renowned for its dedication to the development of innovative therapies for rare diseases. Its flagship product, mitapivat, serves as a testament to the company’s commitment, having already received ODD for pyruvate kinase (PK) deficiency, thalassemia, and sickle cell disease. In a significant achievement for the company, Agios has successfully marketed the first-in-class PK activator for adults with PK deficiency in the US, signifying a novel, disease-modifying therapy for this rare and chronic haemolytic anaemia.
Besides, the company’s clinical pipeline is robust, with ongoing developments for treatments targeting alpha and beta-thalassemia, sickle cell disease, paediatric PK deficiency, MDS-associated anaemia, and phenylketonuria. Furthermore, Agios is exploring the potential of a preclinical TMPRSS6 siRNA as a new treatment avenue for polycythemia vera.
In a strategic move to bolster its financial foundation and future development projects, Agios entered into an agreement in May 2024, selling its stake in vorasidenib to Royalty Pharma for $905 million. Through this deal, Agios secures a 15% royalty on annual US net sales of vorasidenib up to $1 billion, marking a significant financial milestone for the company and enhancing its capability to innovate and deliver treatments for rare diseases.
The FDA’s orphan drug designation for tebapivat not only marks a significant step forward in the fight against MDS but also reinforces Agios Pharmaceuticals’ position as a leading innovator in the development of treatments for rare diseases. With this designation, Agios is well-placed to continue its mission of transforming the lives of patients facing rare and challenging medical conditions.
