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.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.