The fragile X syndrome (FXS) market across the US and Germany is projected to experience explosive growth, quadrupling from $28.7 million in 2025 to $111.9 million by 2030, a CAGR of 31.3% (2025-2030), driven by the anticipated launch of two high-priced targeted therapies for FXS in 2027, according to GlobalData, a leading data and analytics company.
GlobalData’s latest report, “Fragile X Syndrome: Opportunity Assessment and Forecast -Update,” anticipates a stable market until the projected US launches of Harmony Biosciences’ Zygel (cannabidiol) and Shionogi Inc’s zatolmilast in 2027. Zygel is expected to launch in Germany in 2028, whereas zatolmilast is not anticipated to launch in Germany during the forecast period. These therapies are set to represent the first treatments to be indicated for FXS.
Lorraine Palmer, Pharma Analyst at GlobalData, comments: “The introduction of Zygel and zatolmilast could mark a turning point in FXS treatment as, for the first time, there could be therapies offering the potential to address the underlying mechanisms of the disease, a significant unmet need.”
There are currently no approved therapies available for FXS; prescribed treatment consists exclusively of off-label drugs that target individual symptoms of the disease. Examples of such interventions include SSRIs for depressive symptoms and anxiety; stimulants like methylphenidate for hyperactivity, inattention, and impulsivity; antipsychotic medications for aggression; and anticonvulsant agents for seizures.
In addition, according to key opinion leaders (KOLs) interviewed by GlobalData, the behavioral symptoms of irritability, aggression, and anxiety are not adequately addressed by the available treatment options, offering only partial relief. Furthermore, these treatments often come with the burden of side effects, particularly sedation, which can limit normal activity and impact the quality of life of patients. KOLs emphasized the need for therapies that address the underlying etiology of FXS.
The US FXS market, which currently accounts for 96.5% of the combined sales in the US and Germany, is projected to reach $108.3 million by 2030. The German market is expected to grow to $3.7 million by 2030, driven by the launch of Zygel. While the anticipated high costs of Zygel and zatolmilast may be a barrier to their uptake, GlobalData still expects uptake of the agents due to their mechanism of action having the potential to address processes implicated in FXS pathogenesis. KOLs interviewed by GlobalData emphasized that the availability of therapies targeting the underlying etiology of FXS is a key unmet need within the market.
Palmer adds: “Despite the promise of these new therapies, the clinical heterogeneity of FXS suggests that efficacy may vary among patients. This underscores the need for the continued research and development of therapies targeting the underlying etiology of FXS. This need might be met in the future, as currently 73.3% of FXS pipeline agents are in Phase I and II stages of development and most seek to target underlying processes leading to FXS symptoms.”
GlobalData’s report also highlighted the growing prevalence of FXS, with diagnosed cases in the US and Germany expected to go from 69,942 in 2020 to 73,216 in 2030, an AGR of 0.47%. This will be driven by the increasing US total population.
Palmer concludes: “While there previously hasn’t been much movement in the US and German FXS markets, together they are on the cusp of a major transformation and the face of these markets will change within the next five years. The arrival of targeted therapies offers hope for significant improvements in the lives of individuals with FXS.”
