This Market Spotlight report covers the Fragile X Syndrome market, comprising key pipeline drugs, clinical trials, recent events and analyst opinion, key upcoming events, probability of success, a discussion on disease epidemiology, and licensing and asset acquisition deals.

Key Takeaways

• Based on cohorts of children with special educational needs, the prevalence of fragile X syndrome (FXS) is estimated to be 1 in 4,000 in males, and 1 in 8,000 in females.
• The largest proportion of industry-sponsored drugs in active clinical development for FXS are in Phase III. Therapies in active clinical development for FXS focus on targets such as cannabinoid-1/cannabinoid-2 receptor, phosphodiesterase 4, GABA receptors, insulin-like growth factor-1 receptor, NMDA glutamate receptor, and potassium channels. The majority of these pipeline drugs are administered via the oral route.
• The only high-impact upcoming event for drugs in the FXS space is topline Phase III trial results for Zygel. The overall likelihood of approval of a Phase I neurodevelopmental disorders asset is 4.3%, and the average probability a drug advances from Phase III is 25%. Drugs, on average, take 11.8 years from Phase I to approval, compared to 10.3 years in the overall neurology space.
• The distribution of clinical trials across Phase I–IV indicates that the majority of trials for FXS have been in the early and midphases of development, with 88% of trials in Phase I–II, and only 12% in Phase III–IV.
• The US has a substantial lead in the number of FXS clinical trials globally. France leads the major European markets, while Israel has the top spot in Asia.
• Clinical trial activity in the FXS space is dominated by completed trials. Roche has the highest number of completed clinical trials for FXS, with 10 trials.
• Novartis and Roche lead industry sponsors with the highest overall number of clinical trials for FXS.