Indication name: Fragile X syndrome
Fragile X syndrome – Market outlook, Epidemiology, Market Forecast and
Competitive Landscape Report – 2020 To 2030 (Markets covered: US, Germany,
France, Spain, United Kingdom, Italy, Japan & China).
Fragile
X syndrome is a genetic
condition that causes a range of developmental problems including learning
disabilities and cognitive impairment. Usually, males are more severely
affected by this disorder than females. FXS is caused by the transcriptional
silencing of the FMR1 gene (Xq27.3) due to the progressive expansion and
subsequent methylation of (CGG)n trinuleotide repeats in the 5'-untranslated
region of the gene. These full mutations originate from unstable alleles called
premutations (55-200 CGG repeats). In some rare cases, FXS was shown to result
from intragenic FMR1 point mutations or deletions. FMR1 codes for the FMRP, an
RNA-binding protein that regulates protein synthesis and other signaling
pathways in neuronal dendrites. FMR1 silencing is thought to reduce synaptic
plasticity and modulation throughout the brain including the hippocampus.
The Three Fragile X-Associated Disorders
Fragile X Syndrome
Fragile X-Associated Tremor/Ataxia Syndrome
Fragile X-Associated Primary Ovarian Insufficiency
Epidemiology- Thelansis estimates, fragile X syndrome affects about 1 in
4,000 males and 1 in 6,000 to 8,000 females in the USA; that is, it affects
about twice as many males as it does females. However, about four times as many
females appear to be carriers of the altered gene as do males (1:250 females
and 1:1000 males).
Competitive landscape of Fragile X syndrome includes country specific
approved as well as pipeline therapies. Any asset/ product specific designation
or review and Accelerated Approval are being tracked and supplemented with
analyst commentary.
KOLs insights of Fragile X syndrome across 8 MM market from center of
Excellence/ Public/ Private hospitals participated in the study. Insights
around current treatment landscape, epidemiology, clinical characteristics,
future treatment paradigm and Unmet needs.
Fragile X syndromeMarket Forecast: Patient Based Forecast Model (MS.
Excel Based Automated Dashboard) which Data Inputs with sourcing, Market Event
and Product Event, Country specific Forecast Model, Market uptake and patient
share uptake, Attribute Analysis, Analog Analysis, Disease burden and pricing
scenario, Summary and Insights.
S. No Asset Company Partner Phase
1 Zygel Zynerba Pharmaceuticals, Inc. N/A III
2 BPN14770 Tetra Therapeutics Shionogi II
3 Ganaxolone Marinus Pharmaceuticals, Inc. Ligand II
4 OV101 Ovid Therapeutics, Inc. Lundbeck II
5 AUT00206 Autifony Therapeutics Limited Boehringer Ingelheim I
6 Abilify (Oral and
IM) Otsuka Holdings Co., Ltd. Ligand; Lundbeck Investigator Initiated
7 Campral Merck KGaA Allergan Investigator Initiated
8 Zoloft Pfizer Inc. N/A Investigator Initiated
9 MDX Arcturus Therapeutics Ltd. N/A Development Outside U.S.
10 Acamprosate Aclaris Therapeutics, Inc. AOP Orphan Pharmaceuticals Preclinical
11 ANAVEX 2-73 Anavex Life Sciences Corp. N/A Preclinical
12 Bryostatin Neurotrope, Inc. N/A Preclinical
13 DgkK Gene Therapy
Program (Lysogene) Lysogene
SA N/A Preclinical
14 GWP42006 GW Pharmaceuticals plc Otsuka Preclinical
15 NNZ-2591 Neuren Pharmaceuticals Limited N/A Preclinical
16 p70S6K Inhibitor
(Epigen) Epigen Biosciences,
Inc. N/A Preclinical
17 PAK Inhibitor Roche Holding AG Afraxis Preclinical
18 SOL101 Sentinel Oncology Limited N/A Preclinical
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