New Agios Pharma MDS Treatment Gains FDA 'Orphan Drug' Status

Agios Pharmaceuticals has reached a significant milestone in the development of their new myelodysplastic syndrome (MDS) treatment, Tebapivat, with the FDA’s recent designation of the drug as an ‘orphan drug’. Tebapivat has shown promise in treating lower-risk MDS, which affects between 75,000 and 80,000 patients in the US and EU each year. The ‘orphan drug’ status facilitates several benefits for Agios, including additional tax credits, FDA fee exemptions, and the potential for a seven-year market exclusivity period post-approval.

Agios’ Chief Medical Officer, Dr. Sarah Gheuens, emphasized the significance of this designation after Agios wrapped the Phase 2a study of Tebapivat; with the company now proceeding with Phase 2b of the trial.

The OxyDial team is closely following Agios’ progress, with this designation not only highlighting the company’s dedication to fighting rare hematologic diseases, but also helping set the stage for the potentially transformative impacts on disease management and patient care.