Agios Pharmaceuticals has announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to tebapivat (AG-946) to treat myelodysplastic syndromes (MDS).
The development comes after the company completed a Phase IIa trial of tebapivat in lower-risk MDS in 2023 and is now initiating a Phase IIb study of the asset for the same indication.
The FDA's Office of Orphan Drug Products awards ODD to aid the development of treatments for rare diseases affecting fewer than 200,000 people in the US.
The designation offers several benefits to Agios, including tax credits, FDA fee exemptions and the potential for seven years of market exclusivity post-approval.
Agios Pharmaceuticals research and development chief medical officer and head Sarah Gheuens stated: “Receiving orphan drug designation for tebapivat in MDS underscores the importance of bringing new oral treatment options to patients suffering from this rare disease.
“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 focuses on developing groundbreaking therapies for patients with rare diseases.
Its lead asset is mitapivat, a PK [pyruvate kinase] activator. This asset previously received ODD for PK deficiency, thalassaemia and sickle cell disease.
In the US, Agios markets a first-in-class PK activator for adults with PK deficiency, offering a novel disease-modifying therapy for this rare and chronic haemolytic anaemia.
Agios' clinical pipeline also includes treatments for alpha and beta-thalassemia, sickle cell disease, paediatric PK deficiency, MDS-associated anaemia, and phenylketonuria.
Agios is developing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera.
In May 2024, Agios agreed to sell its stake in vorasidenib to Royalty Pharma for $905m.
The company, which owns the royalties on the drug’s US sales, receives a 15% royalty on annual US net sales of vorasidenib up to $1bn.
