ImmuneSensor Therapeutics Receives Orphan Drug and Rare Pediatric Disease Designations from the U.S. FDA for cGAS Inhibitor Drug Candidate, IMSB301, for the Treatment of Aicardi Goutières Syndrome (AGS)

Aicardi-Goutières syndrome (AGS)

https://www.immunesensor.com/news/112024

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for ImmuneSensor Therapeutics drug candidate IMSB301 for Aicardi Goutières Syndrome (AGS) which is currently in an ongoing Phase 1 trial. ODD was introduced to encourage the development of drugs to treat conditions affecting fewer the 200,000 people in the US and offers a number of benefits to the pharmaceutical company as well as potentially allowing for priority review. The Rare Paediatric Disease designation offers similar benefits but is concerned with the development of drugs for diseases affecting a small number of children under the age of 18. This program intends to help with the development of new drugs intended to prevent or treat rare paediatric diseases. These are important steps for the development of this treatment for AGS.