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
Related News
-
News Swiss pharma industry warns US tariffs could harm global patient care
The Swiss pharmaceutical sector has raised alarms over new US tariffs on medicines, warning of disrupted supply chains, increased costs, and delayed innovation, all of which could jeopardise patient access to essential treatments worldwide.
-
News Understanding the Benefits and Advances of Cleanroom Technology
In an industry where precision and sterility are crucial concerns, cleanrooms play a vital role in maintaining the integrity of pharmaceutical products like drugs, vaccines, and other medical products. So, what is a cleanroom?
-
News From Pharma to Table? Direct-to-consumer pharma sales and price cuts ramp up in US
The pharmaceutical industry is witnessing a seismic shift as major companies embrace direct-to-consumer sales models and announce significant price reductions across the United States, responding to mounting pressure from the Trump administration to ta... -
News A Day in the Life of a Director of Business Development
In this Day in the Life of interview, Thomas Hauser, Director of Business Development at Tjoapack. Thomas has spent nearly 30 years in the pharmaceutical industry and describes how he still finds curiosity and excitement in the bigger picture of global... -
.png)
News Women in Pharma: Our hopes for 2025 and beyond
Our last instalment for 2024 of the Women in Pharma series brings you messages direct from the Informa Markets CPHI team as they discuss the advice and insights they have carried throughout their roles working at CPHI, and what they hope to see for the... -
News CPHI Milan 2024 - From the Floor
Milan and CPHI welcome you to 2024 CPHI Milan! As we celebrate the 35th edition of our flagship CPHI show, editors Vivian Xie and Lucy Chard bring you the latest from the show floor, conference sessions, and innovative solutions from all exhibitors, at... -
News The BIOSECURE Act: implications for the pharma supply chain
On September 9, 2024, the US House of Representatives voted to pass the bill titled the BIOSECURE Act (the Act), which lists several Chinese companies in the pharmaceutical supply chain. The Act will prohibit American companies from contracting or doin... -
News A Day in the Life of a Global CDMO Chapter Lead – Manufacturing
The 'Day in the Life of' series has covered many aspects of the pharmaceutical pipeline, including R&D and procurement, now we're taking a look at manufacturing from a global CDMO perspective.