FDA has granted orphan drug designation (ODD) to tebapivat (AG-946), a novel pyruvate kinase (PK) activator being developed by Agios Pharmaceuticals, for the treatment of myelodysplastic syndromes (MDS).
Sarah Gheuens, CMO at Agios, acknowledged the FDA’s ODD for tebapivat in MDS as a significant milestone.
She emphasized the importance of new oral treatments for this rare disease, noting that tebapivat aims to be the first oral therapy for anemia in lower-risk MDS, which affects 75,000-80,000 patients in the U.S. and EU5.
Agios completed a Phase 2a trial of tebapivat in lower-risk MDS, demonstrating good tolerability and promising improvements in blood cell counts. Based on these results, Agios launched a larger Phase 2b trial.
The FDA’s ODD provided Agios with specific incentives, including tax credits, exemptions from certain FDA fees, and the potential for seven years of market exclusivity following tebapivat approval.
Agios is committed to advancing the development of tebepivat and other potential therapies for patients with MDS and other rare blood cancers.
Source: Agios Announces FDA Orphan Drug Designation Granted to Tebapivat (AG-946) for Treatment of Myelodysplastic Syndromes (MDS)
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