Agios Pharma's tebapivat earns FDA orphan drug status

Agios Pharmaceuticals has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for their drug tebapivat (AG-946) to treat myelodysplastic syndromes (MDS). This significant milestone follows the completion of a Phase IIa trial for tebapivat in lower-risk MDS earlier in 2023, and the company is now moving forward with a Phase IIb study for the same condition.

ODD is granted by the FDA’s Office of Orphan Drug Products to promote the development of treatments for rare diseases, which typically affect fewer than 200,000 people in the United States. This designation is crucial for Agios as it provides various benefits, including tax credits, exemptions from FDA fees, and potentially seven years of market exclusivity once the drug gains approval.

Sarah Gheuens, the chief medical officer and head of research and development at Agios Pharmaceuticals, commented on the significance of this designation. She emphasized the urgency of introducing new oral treatment options for patients suffering from this rare disease. According to Gheuens, the goal is to offer the first oral therapy that effectively tackles anaemia caused by ineffective erythropoiesis in lower-risk MDS. This condition affects around 75,000 to 80,000 patients across France, Germany, Italy, Spain, the UK, and the US, accounting for roughly 70% of all MDS cases.

Agios Pharmaceuticals is dedicated to creating innovative therapies for patients with rare diseases. Their primary asset, mitapivat, is a pyruvate kinase (PK) activator that has already received ODD for treating PK deficiency, thalassemia, and sickle cell disease. In the US, Agios has launched a first-in-class PK activator for adults with PK deficiency, providing a novel approach to treating this rare and chronic haemolytic anaemia.

The company's clinical pipeline includes potential treatments for a range of conditions, such as alpha and beta-thalassemia, sickle cell disease, paediatric PK deficiency, MDS-associated anaemia, and phenylketonuria. Additionally, Agios is in the preclinical stages of developing a TMPRSS6 siRNA treatment for polycythemia vera.

In a recent strategic move, Agios agreed in May 2024 to sell its stake in vorasidenib to Royalty Pharma for $905 million. Under the agreement, Agios, which holds the royalties on the drug's US sales, will receive a 15% royalty on annual US net sales of vorasidenib up to $1 billion. This transaction reflects Agios' ongoing strategy to focus on its core strengths and continue advancing its promising pipeline of therapies for rare diseases.