Transthyretin Amyloidosis – Market outlook, Epidemiology, Competitive Landscape and Market Forecast Report – 2020 To 2030


 The hereditary form of ATTR is caused by autosomal dominant mutations in the TTR gene. Amyloid formation associated with the amyloidogenic mutations associated with destabilization and dissociation of the TTR tetramer, leading to abnormally folded monomers that ultimately self-assemble to amyloid fibrils. These TTR amyloid fibrils are then deposited extracellularly in various tissues. These protein deposits most frequently occur in the peripheral nervous system, which is made up of nerves connecting the brain and spinal cord to muscles and sensory cells that detect sensations such as touch, pain, heat, and sound.

 

Protein deposits in these nerves result in a loss of sensation in the extremities (peripheral neuropathy). The autonomic nervous system, which controls involuntary body functions such as blood pressure, heart rate, and digestion, may also be affected by amyloidosis. In some cases, the brain and spinal cord (central nervous system) are affected.

 

Therapies like Vyndaqel and Vyndamax from Pfizer have gained US FDA approval for the treatment of wild-type or hereditary ATTR-CM. Emerging therapies that stabilize TTR have been shown to improve outcomes for patients with ATTR-CM, and TTR silencer therapies are entering late-phase clinical trials.

 ATTR is a rare indication with incidence rates varying with geographies and ancestry. The indication is less prevalent among Caucasians where it is estimated to affect 1 to 2 cases in 100,000 population.

 

The competitive landscape of Transthyretin Amyloidosis 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 Transthyretin Amyloidosis across 8 MM market from the centre of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.

Transthyretin Amyloidosis 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.

Read more: Transthyretin Amyloidosis – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030

S. No    Asset               Company                                 Stage

1          NTLA-2001       Intellia Therapeutics      Phase 1

2          Vutrisiran          Alnylam Pharmaceuticals           Phase 3

3          AKCEA-TTR-LRx           Akcea Therapeutics       Phase 3

4          SOM0226         SOM Biotech SL            Phase 2

5          ALXN2060        Alexion Pharmaceuticals            Phase 3

6          Tolcapone         Corino Therapeutics, Inc.           Phase 1

7          Tafamidis          Pfizer    Phase 3

8          NI006   Neurimmune AG           Phase 1

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