ConSynance earns FDA nod for Prader-Willi treatment

The US Food and Drug Administration (FDA) has granted ConSynance Therapeutics a rare pediatric disease designation for its oral therapy targeting Prader-Willi syndrome. This designation enables the company to receive a priority review voucher if the FDA approves its drug candidate, CSTI-500. These vouchers are valuable, as they reduce FDA review time by four months, expediting a drug's market entry. Companies can either use these vouchers for their own drugs or sell them, with recent transactions averaging around $100 million.

CSTI-500 is aimed at treating Prader-Willi syndrome in children and adolescents. This condition is marked by neuropsychiatric behaviors such as an intense desire for excessive eating and frequent temper outbursts. CSTI-500 is a triple monoamine reuptake inhibitor (TRI) that helps restore serotonin, dopamine, and norepinephrine levels, which are linked to these symptoms. According to ConSynance, the drug is the first of its kind to address these specific challenges associated with Prader-Willi syndrome. The company plans to initiate a Phase II study next year.

Current treatments for children with Prader-Willi syndrome include human growth hormones, which can enhance muscle size and strength, boost energy levels, and alter facial appearance. The syndrome is also associated with behavioral difficulties, poor muscle development, learning challenges, restricted growth, and underdeveloped sexual characteristics.

The National Organization for Rare Disorders (NORD) estimates that the incidence rate of Prader-Willi syndrome ranges between one in 10,000 to one in 30,000 people in the general population.

Position emission tomography (PET) scans have shown CSTI-500's effectiveness in targeting brain activity in healthy volunteers. The drug has been tested on nearly 100 individuals across three Phase I clinical trials. Two of these trials involved single and multiple ascending doses in healthy volunteers, while the third trial (NCT05504395) included ten patients with Prader-Willi syndrome. There were no severe adverse events reported, and the drug showed consistent efficacy across all three trials.

Shuang Liu, CEO of ConSynance, emphasized the significance of CSTI-500, stating, "CSTI-500 stands out as the first drug candidate for Prader-Willi syndrome designed to address both hyperphagia and severe temper outbursts, providing a potential breakthrough and comprehensive treatment solution for this challenging disorder."