Indication name: Hemophilia B
Hemophilia B – Market outlook,
Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030
(Markets covered: US, Germany, France, Spain, United Kingdom, Italy, Japan
& China)
Hemophilia B (also called Christmas
Disease) is a rare hereditary bleeding disorder caused by the insufficient
level of blood protein called coagulation factor IX. According to Thelansis
disease modeling data, about 30% of new cases of hemophilia B is caused by
mutation in the F9 gene, responsible for the formation of factor IX protein.
This mutation is due to X-linked recessive disorder which alter genes on the X
chromosome.
There is another unusaul form of
factor IX deficiency called Hemophilia B Leyden approximately 3% of the
hemophilia B cases. Depending upon the particular hemophilia B Leyden mutation
present, there are undetectable levels of factor IX present early in life that
increase over time. By midlife, these patients have factor IX levels at the low
end of the normal range.
According to Thelansis,
Hemophilia B affects 1 in 500 male births in the U.S., and approximately 100
babies are born with hemophilia B each year. Approximately 400,000 people worldwide
are living with hemophilia, and about 24,000 are living with it in the United
States alone.
Classification- Hemophilia B is
classified as mild, moderate or severe based upon the activity level of factor
IX. In mild cases, bleeding symptoms may occur only after surgery, injury or a
dental procedure. In some moderate and most severe cases, bleeding symptoms may
occur after a minor injury or spontaneously, meaning without an identifiable
cause.
KOLs
insights of Hemophilia B 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.
Competitive landscape of
Hemophilia B 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.
Hemophilia B Market 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 Stage
1 rFIXFc Bioverativ Therapeutics Inc. Phase 1
2 Tranacogene Dezaparvovec (AMT-061) UniQure Phase 3
3 AskBio009 Baxalta now part of Shire Phase 2
4 FLT180a Freeline Therapeutics Phase 3
5 rIX-FP CSL Behring Phase 3
6 IB1001 Aptevo BioTherapeutics Phase 3
7 BeneFIX Grifols Biologicals, LLC Phase 2
8 PF-06838435/ fidanacogene
elaparvovec Pfizer Phase 3
9 MarzAA Catalyst Biosciences Phase 3
10 APVO101 Medexus Pharma, Inc. Phase 3
11 IB1001 Cangene Corporation Phase 3
12 nonacog beta pegol Novo Nordisk A/S Phase 3
13 Coagulation Factor VIIa
(Recombinant) rEVO Biologics Phase 3
14 Coagulation FVIIa (Recombinant) LFB USA, Inc. Phase 3
15 SerpinPC ApcinteX Ltd Phase 2
16 MOD-5014 OPKO Health, Inc. Phase 2
17 BAY1093884 Bayer Phase 1
18 ALN-AT3SC Alnylam Pharmaceuticals Phase 1
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