Agios Gets FDA Orphan Designation for Tebapivat in Myelodysplastic Syndromes

By Colin Kellaher

Agios Pharmaceuticals has won Food and Drug Administration orphan-drug designation for its proposed tebapivat treatment for a group of rare blood cancers.

Agios on Wednesday said the designation covers tebapivat for the treatment of myelodysplastic syndromes, or MDS, in which immature blood cells in the bone marrow don't mature or become healthy blood cells.

The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S. and provides for an extended marketing exclusivity period against competition.

The Cambridge, Mass., biopharmaceutical company completed a Phase 2a study of tebapivat in lower-risk MDS late last year and is currently initiating a Phase 2b study.

Write to Colin Kellaher at colin.kellaher@wsj.com

(END) Dow Jones Newswires

September 11, 2024 07:43 ET (11:43 GMT)

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