UK-based drug discovery company Summit has received the US Food & Drug Administration's (FDA) orphan drug status for SMT C1100, used to treat Duchenne muscular dystrophy.
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 executive chairman Barry Price said the company 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 will provide additional regulatory support and various commercial benefits including extended periods of market exclusivity," Price said.