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news.The US Food and Drug Administration (FDA) has granted fast track designation for Catabasis Pharmaceuticals' CAT-1004, an oral small molecule, to treat duchenne muscular dystrophy (DMD).
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CAT-1004 inhibits activated NF-kB, a protein that coordinates cellular response to muscular damage, stress and inflammation and plays a major role in muscle health.
DMD involves progressive muscle degeneration that eventually leads to death and for which there are no approved therapies in the US.
Catabasis co-founder and chief executive officer Jill Milne said: "Fast Track designation for CAT-1004 highlights its potential to treat a serious, life threatening disease with few treatment options for these young patients.
"By targeting activated NF-kB in pre-clinical studies, CAT-1004 has demonstrated disease-modifying potential for this devastating condition."
The company noted that in Phase I clinical trials, CAT-1004 inhibited activated NF-kB and was well-tolerated with no observed safety concerns.
Earlier, the FDA has also granted orphan drug designation to CAT-1004 for the treatment of DMD.
Catabasis is a US-based clinical-stage drug development company built on a pathway pharmacology technology platform.