Promising pipeline drugs to transform Fragile X Syndrome treatment market by 2030: GlobalData

The latest report from GlobalData has highlighted that promising pipeline drugs Zygel, zatolmilast, trofinetide and acamprosate calcium SR are set to offer new treatment options for people with Fragile X Syndrome (FXS)—a rare genetic disorder that is the leading cause of inherited intellectual disability. These products will help propel the FXS market and will add $23.3 million to the market by 2030.

Christie Wong, Neurology Analyst, GlobalData, commented, “…….GlobalData expects Zynerba Pharmaceuticals’ Zygel will be the first to market, with a primary completion date for its phase-III trial expected in September 2023. Zygel could offer an important therapeutic option for patients who are the most severely impacted with FXS and could bring in sales of $18.1 million by 2030.

“As well as Zygel, the upcoming release of Tetra Therapeutics’ zatolmilast, Neuren Pharmaceuticals’ and Acadia Pharmaceuticals’ trofinetide, and Confluence Pharmaceuticals’ acamprosate calcium are expected to transform the market, boasting diverse mechanisms of action (MOA). Together, these will help create a new dynamic market with a variety of options for FXS patients.”

FXS is a life-long disorder that causes a range of developmental and behavioural challenges including learning disabilities and cognitive impairment. Unfortunately, there is no cure or approved therapies available for FXS patients. This leaves patients reliant on the use of off-label products that target individual symptoms of the disease.

Wong continued, “The current FXS treatments consist mainly of genericised drugs, which are used to target psychiatric and neurological symptoms. Examples include selective serotonin reuptake inhibitors for depressive symptoms and anxiety; stimulants such as methylphenidate for hyperactivity, inattention and impulsivity; antipsychotic medication for aggression, and anticonvulsant agents for seizures.

“There is a need for novel drugs with improved safety and efficacy profiles as many FXS patients are being treated with a number of different drugs to help alleviate their individual sets of symptoms. Furthermore, many of the currently used drugs have a boxed warning due to the high risk of abuse potential and dependence.

“Therapies that target specific molecular mechanisms involved in the pathogenesis of FXS are considered the holy grail of drug development for the disease. The pipeline agents for FXS demonstrate a variety of MOAs and are expected to expand the FXS treatment options in the US and Germany.”