Indication name: Friedreich
ataxia (FA)
"Friedreich ataxia (FA) – Market
outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020
To 2030
Friedreich
ataxia (FA) is a rare inherited disease that causes progressive nervous system
damage and movement problems. It usually
begins in childhood and leads to impaired muscle coordination (ataxia) that
worsens over time. The gene responsible for FRDA has been designated FXN. The
FXN gene codes for frataxin, a protein that is required for proper functioning
of mitochondria, which are the energy producing parts of our cells. In most
affected individuals, the FXN gene contains a very specific type of error
called an expanded GAA trinucleotide repeat.
Epidemiology-
Based on epidemiology study of Thelansis
18,660 patients are likely to be diagnosed patient in 6 Major markets (USA and
EU5), out of which 16,800 patients will able to meet the treatment for the year
2021 for the same 6 major markets.
Geography Covered
North America- United States and Canada
Europe- EU5 (Germany, France, Italy, Spain,
and the United Kingdom)
Other countries- Japan & China
Study Period: 2020-2030
Competitive landscape of Friedreich ataxia
(FA) 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 Friedreich ataxia (FA)
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.
Friedreich ataxia (FA) Market Forecasting: 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 Stage
1
Omaveloxolone Capsules
Reata Pharmaceuticals, Inc.
Phase 2
2
Vatiquinone PTC
Therapeutics Phase 3
3
deferiprone ApoPharma Phase 2
4
cohort Retrotope, Inc. Phase 2
5
MIB-626 Metro International
Biotech, LLC Phase 2
6
CTI-1601 Larimar
Therapeutics, Inc. Phase 1
7
MIN-102 Minoryx
Therapeutics, S.L. Phase 2
8
TAK-831 Takeda Phase 2
9
interferon γ-1b Horizon
Pharma Ireland, Ltd., Dublin Ireland
Phase 3
10
Idebenone Santhera
Pharmaceuticals Phase 3
11
VP 20629 Shire Phase 1
12
Lu AA24493 H. Lundbeck
A/S Phase 2
13
alpha-tocopherolquinone (A0001)
Penwest Pharmaceuticals Co.
Phase 2
14
EGb 761 120 mg Ipsen Phase 2
15
(+)-Epicatechin Cardero
Therapeutics, Inc. Phase 2
16
EPI-743 Edison
Pharmaceuticals Inc Phase 2"
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