On August 4th, Roche Pharmaceuticals announced that their huntingtin-lowering therapy was granted PRIME status from the European Medicines Agency (EMA). The EMA is the European equivalent of the United States Food and Drug Administration (FDA). In Europe, PRIME (PRIority MEdicines) designation means that the EMA has agreed that this drug, RG6042 (formerly IONIS-HTTRx) could potentially fill an urgent need to combat HD. For this reason, the EMA will make it a priority to evaluate any future clinical findings at a faster rate.
PRIME status does not mean that the drug is now available to the public. But it’s exciting nonetheless – it’s a commitment from the EMA to prioritize HD. This means that the clinical trial approvals and eventual data review will move faster, once there is more information on effectiveness in humans. The Phase III trial of this drug will take place as planned, and we are cautiously optimistic that it will begin by the end of the year, in both Europe and the USA. HDSA still does not have information about what exactly the trial will look like, including specific sites and timing, but we will continue to work closely with Roche to share any information across our networks as it becomes available.
Milton Wexler Celebration of Life: Boston HD Conference
Every other August, the Hereditary Disease Foundation holds an invited research conference to bring together scientists studying Huntington’s disease and related neurological disorders. Dr. Leora Fox and Dr. George Yohrling are headed to Boston this week for the 2018 conference, to make connections, hear the latest in cutting-edge HD research, and participate in three days of shared HD science from around the world.
Berman-Topper Fellow blogs about research progress
2016 HDSA Berman-Topper Fellow Dr. Tamara Maiuri blogs regularly in real time about her research progress. This week, check out her latest post!