Global Women Member, distinguished professor director of medicinal chemistry at Waipapa Taumata Rau, University of Auckland, Dame Margaret Brimble, is about to make history.
She and her team are one step away from bringing a breakthrough drug for Rett Syndrome to light.
20 years of research, and unwavering dedication has resulted in the formulation of Trofinetide, the first ever drug treatment for Rett Syndrome. Promisingly, it has been granted final approval by the US Food and Drug Administration (FDA).
Adding to the milestones is the fact that it is the first New Zealand-discovered neurological drug to win FDA approval.
“It’s a horrible disease, and finally there’s a treatment. We’ve waited so long for this, it’s a great day” — Dame Margaret Brimble
“I’m over the moon for all of the families who need the drug,” shares Dame Margaret, director of medicinal chemistry at Waipapa Taumata Rau, University of Auckland. “It’s a horrible disease, and finally there’s a treatment. We’ve waited so long for this, it’s a great day.”
There’s a gendered lens to this research and condition: Rett Syndrome almost exclusively impacts females. It’s a condition where motor and communication skills stall or regress in the early years of a child’s life. Success in Trofinetide’s clinical trials, however, have resulted in young girls talking or walking for the first time in their life.
Dame Margaret’s momentum shows no signs of slowing down
Dame Margaret’s momentum shows no signs of slowing down. A second neurological drug called NNZ-2591 from Dame Margaret and her team working with Dr Jian Guan, officially has approval to enter phase 2 clinical trials for the treatment of Phelan-McDermid syndrome, Angelman syndrome, Pitt-Hopkins syndrome and Prader-Willi syndrome.
Listen to Dame Margaret speak about this incredible milestone on Newstalk ZB, or get the full details online here.