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Market Spotlight: Fragile X Syndrome

  • ID: 4775087
  • Report
  • January 2020
  • Region: Global
  • 26 Pages
  • Datamonitor Healthcare
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This Market Spotlight report covers the Fragile X Syndrome market, comprising key pipeline drugs, clinical trials, recent events and analyst opinion, upcoming events, patent information, 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, prevalence of fragile X syndrome (FXS) is estimated to be 1 in 4,000 in males, and 1 in 8,000 in females.
  • The majority of industry-sponsored drugs in active clinical development for FXS are in Phase II, with only one drug in Phase III. Therapies in active clinical development for FXS focus on targets such as cannabinoid-1/cannabinoid-2 receptor, phosphodiesterase 4, potassium channels, and GABA-A receptor. The majority of the pipeline drugs are administered via the oral route, with two drugs being tested in intravenous and topical formulations.
  • High-impact upcoming events for drugs in the FXS space comprise topline Phase III trial results for Zygel, and topline Phase II trial results for OV101 and BPN14770.
  • The overall likelihood of approval of a Phase I neurodevelopmental disorders asset is 4.2%, and the average probability a drug advances from Phase III is 28.6%. Drugs, on average, take 8.8 years from Phase I to approval, compared to 9.8 years in the overall neurology space.
  • There have been five licensing and asset acquisition deals involving FXS drugs during 2015-20. The $737.5m exclusive licensing agreement made in 2017 between Boehringer Ingelheim and Autifony Therapeutics for a collaboration on a novel therapeutic approach to a range of CNS disorders was the largest deal.
  • All clinical trials for FXS have been in Phase II, with no Phase III trials to date.
  • The US leads in terms of the number of FXS clinical trials globally.
Note: Product cover images may vary from those shown
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Key Takeaways

Disease Background


  • Premutation
Pipeline Drugs

Recent Events and Analyst Opinion
  • Campral for FXS (June 25, 2019)
Key Upcoming Events

Probability of Success

Licensing and Asset Acquisition Deals

Parent Patents

Clinical Trial Landscape
  • Sponsors by status
  • Sponsors by phase


List of Figures
Figure 1: Overview of pipeline drugs for FXS in the US
Figure 2: Pipeline drugs for FXS, by company
Figure 3: Pipeline drugs for FXS, by drug type
Figure 4: Pipeline drugs for FXS, by classification
Figure 5: Key upcoming events in FXS
Figure 6: Probability of success in the FXS pipeline
Figure 7: Licensing and asset acquisition deals in FXS, 2015–20
Figure 8: Parent patents in FXS
Figure 9: Clinical trials in FXS
Figure 10: Top 10 drugs for clinical trials in FXS
Figure 11: Top 10 companies for clinical trials in FXS
Figure 12: Trial locations in FXS
Figure 13: FXS trials status
Figure 14: FXS trials sponsors, by phase

List of Tables
Table 1: Pipeline drugs for FXS in the US
Table 2: Campral for FXS (June 25, 2019)
Note: Product cover images may vary from those shown
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