The FDA has granted Rare Pediatric Disease Designation (RPDD) to ConSynance Therapeutics' new investigational drug, CSTI-500 for the treatment of Prader-Willi Syndrome in children and adolescents. This special status is granted to drugs and biologics intended to treat rare diseases, affecting fewer than 200,000 people in the United States, that are serious or life-threatening and primarily impact those under 18 years of age.
Receiving RPDD does not change the regulatory requirements for approval, but it can provide a significant advantage in terms of an expedited review timeline.
“Receiving RPDD from the FDA is a significant regulatory milestone for ConSynance as we prepare for the Phase 2 study of CSTI-500 for PWS,” said Shuang Liu, PhD, Founder and CEO of ConSynance.
CSTI-500 is a phase 2-ready, first-in-class, orally administered Triple Monoamine Reuptake Inhibitor (TRI). It is designed to optimally increase the levels of three critical neurotransmitters, serotonin, dopamine, and norepinephrine, in the synaptic clefts of neurons. CSTI-500 has demonstrated brain target engagement in healthy volunteers.
These data, along with the corresponding pharmacokinetics data, predict the efficacy of CSTI-500 in PWS and other neuropsychiatric indications. CSTI-500 has been studied in nearly 100 humans across three Phase 1 clinical trials, including 10 PWS patients, setting the stage for a Phase 2 study expected to commence in 2025.
Read Consynance's full press release here.
