+353-1-416-8900REST OF WORLD
+44-20-3973-8888REST OF WORLD
1-917-300-0470EAST COAST U.S
1-800-526-8630U.S. (TOLL FREE)

PRINTER FRIENDLY

Market Spotlight: Fragile X Syndrome

  • ID: 4775087
  • Report
  • October 2020
  • Region: Global
  • 30 Pages
  • Datamonitor Healthcare
This Market Spotlight report covers the Fragile X Syndrome market, comprising key pipeline drugs, clinical trials, recent events and analyst opinion, 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, 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, GABA-A receptor, insulin-like growth factor-1 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 II trial results for BPN14770. The overall likelihood of approval of a Phase I neurodevelopmental disorders asset is 3.3%, and the average probability a drug advances from Phase III is 22.2%. Drugs, on average, take 8.2 years from Phase I to approval, compared to 9.8 years in the overall neurology space.

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.

No company has conducted more than one trial in the FXS space
Note: Product cover images may vary from those shown
OVERVIEW

KEY TAKEAWAYS

DISEASE BACKGROUND

TREATMENT

EPIDEMIOLOGY
  • Premutation
PIPELINE DRUGS

RECENT EVENTS AND ANALYST OPINION
  • Zygel for FXS (October 15, 2020)
  • Zygel for FXS (June 30, 2020)
  • OV101 for FXS (May 7, 2020)
KEY UPCOMING EVENTS

PROBABILITY OF SUCCESS

LICENSING AND ASSET ACQUISITION DEALS
  • Shionogi To Add CNS Critical Mass With Tetra Buyout
CLINICAL TRIAL LANDSCAPE
  • Sponsors by status
  • Sponsors by phase
BIBLIOGRAPHY

APPENDIX

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: Zygel for FXS (October 15, 2020): Phase III - CONNECT-FX (US, AUS, NZ)
  • Figure 6: Zygel for FXS (June 30, 2020): Phase III - CONNECT-FX (US, AUS, NZ)
  • Figure 7: OV101 for FXS (May 7, 2020): Phase II - ROCKET
  • Figure 8: Key upcoming events in FXS
  • Figure 9: Probability of success in the neurodevelopmental disorders pipeline
  • Figure 10: Clinical trials in FXS
  • Figure 11: Top 10 drugs for clinical trials in FXS
  • Figure 12: Top 10 companies for clinical trials in FXS
  • Figure 13: Trial locations in FXS
  • Figure 14: FXS trials status
  • Figure 15: FXS trials sponsors, by phase
List of Tables
  • Table 1: Pipeline drugs for FXS in the US
  • Table 2: Zygel for FXS (October 15, 2020)
  • Table 3: Zygel for FXS (June 30, 2020)
  • Table 4: OV101 for FXS (May 7, 2020)
  • Table 5: Campral for FXS (June 25, 2019)
Note: Product cover images may vary from those shown
Adroll
adroll