Summit Therapeutics PLC (LON:SUMM NASDAQ:SMMT) new treatment for muscle wasting disease Duchenne muscular dystrophy has been awarded US fast track status
The new treatment, ezutromid had already been granted Orphan Drug designation for serious illnesses that affect relative small numbers of people by the FDA, the US regulator, and the European Medicines Agency.
A US fast track designation expedites the approval of new drugs that treat conditions classified as an unmet medical need. The drug is currently undergoing a phase II trial, PhaseOutDMD.
DMD is a fatal disease that affects around 50,000 boys and young men globally and is caused by faults in the gene that encodes dystrophin, a protein that is essential for the healthy function of all muscles.
Ezutromid is a utrophin modulator and acts as a potential disease-modifying treatment.
"Fast Track designation underscores the importance that the FDA places on developing new treatments for life-threatening disorders, such as DMD, and aligns well with our recently outlined strategy to accelerate the development of ezutromid to market," said Glyn Edwards, Summit’s chief executive.
He added that one ezutromid's advantages is that it has the potential to aid all patients with DMD, regardless of their underlying genetic fault.