Summit Receives FDA Orphan Drug Status for SMT C1100

Summit's DMD therapy has earned the US FDA orphan drug status.

UK-based drug discovery firm Summit Corporation plc has received the US FDA orphan drug status for SMT C1100, used to treat Duchenne muscular dystrophy (DMD).

SMT C1100 is a small molecule drug that enhances production of a similar naturally occurring protein called utrophin to replace the missing dystrophin.

Pre-clinical data has shown that SMT C1100 can improve utrophin production.

Summit plans to commence a clinical study to evaluate a new formulation of the drug that will benefit all patients with the fatal disease.

"Our clinical candidate has now been granted orphan drug status both in Europe and the US, and this status wi