Homozygous Familial Hypercholesterolemia Market: Future Forecast Indicates a Growth Rate at a CAGR of 5.7%, During the Study Period [2017-2030] | DelveInsight

LAS VEGAS, June 15, 2021 /PRNewswire/ -- DelveInsight's "Homozygous Familial Hypercholesterolemia (HoFH) Market" report provides a thorough comprehension of the Homozygous Familial Hypercholesterolemia historical and forecasted epidemiology, and the Homozygous Familial Hypercholesterolemia market trends in the 7MM [the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan]. The HoFH market report also proffers an analysis of the current HoFH treatment algorithm/practice, market drivers, market barriers, and unmet medical needs. 

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Some of the imperative takeaways from the Homozygous Familial Hypercholesterolemia Market Research Report

  • Several key pharmaceutical companies such as Amgen, Aegerion Pharmaceutical, Regeneron Pharmaceuticals, Alnylam Pharmaceuticals, Novartis, Lib Therapeutics, Sanofi, Neurobo Pharmaceuticals, Arrowhead Pharmaceuticals, and others are developing novel products to improve the Homozygous Familial Hypercholesterolemia treatment outlook. 
  • Juxtapid (lomitapide), Repatha (evolocumab), Evkeeza (evinacumab), and Praluent (alirocumab) are the US FDA approved for the HoFH treatment.
  • Statins alone or in combination with ezetimibe are the first line of treatment for HoFH patients, and other drugs are generally given in combination with statins. Other drugs used for treating HoFH include Repatha (PCSK9 Inhibitor), Juxtapid (lomitapide; MTP inhibitor), and others (Bile acid sequestrants (cholestyramine, colesevelam), Stanol esters). Additionally, Lipoprotein Apheresis is the only long-term treatment with the potential to slow early atherosclerosis and prolong survival in HoFH patients. 
  • Recently, Praluent and Evkeeza received approval in adult patients with HoFH; however, Evkeeza's high cost might pose some concern. 
  • Homozygous Familial Hypercholesterolemia market to enlarge because of the raising awareness of the disease, improvement in the diagnosis methodologies, incremental healthcare spending across the world, the launch of potential emerging therapies, demonstrating greater efficacy in a smaller injection volume. Besides, a huge window is open for the key players to develop effective therapies that can achieve more than 50% LDL-C lowering capacity and increase overall patient compliance. 
  • However, hindering factors such as the use of off-label therapies and generics, lack of effective reimbursement policy for HoFH treatment cost, high rate of under-diagnosis and under-treatment, lack of appropriate knowledge among clinicians pose an enormous threat towards the improvement of the overall Homozygous Familial Hypercholesterolemia treatment regimen that will act as a roadblock to the Homozygous Familial Hypercholesterolemia market's growth. 
  • In terms of current therapies, real-world data suggest that combinations of statins with other cholesterol-lowering medications, including bile acid sequestrants, niacin, fibrates, probucol, and LA, are being used successfully in treating HoFH patients and can be considered to further lower LDL-C levels. However, their use may be limited by tolerability and availability. Considering this real-world data, only a slight dip in patient share has been assumed in the forecast model. This assumption has been strengthened because the maximum emerging therapies are being investigated as an adjunct to the currently available standard therapies for HoFH. 

For further information on Market Impact by Therapies, visit: Homozygous Familial Hypercholesterolemia Drugs Market Analysis 

Homozygous Familial Hypercholesterolaemia (HoFH) is a rare and life-threatening disease characterized initially clinically by plasma cholesterol levels >13 mmol/L, extensive xanthomas, and marked premature and progressive atherosclerotic cardiovascular disease (ACVD). It is a more severe form of Familial Hypercholesterolemia (FH), an autosomal dominant genetic disorder.

As per DelveInsight's analysis, the total HoFH prevalence in the 7MM was estimated at around 3,882 cases in 2020. It was also observed that HoFH affects males and females equally. 

The Homozygous Familial Hypercholesterolemia Market Report provides historical as well as forecasted epidemiological analysis segmented into: 

  • Prevalent Population of Homozygous Familial Hypercholesterolemia (HoFH)
  • Diagnosed HoFH Prevalent Population 
  • Mutation-specific Distribution of Homozygous Familial Hypercholesterolemia 

Get a complete epidemiological segmentation @ Homozygous Familial Hypercholesterolemia Epidemiological Analysis 

Homozygous Familial Hypercholesterolemia Treatment Market 

Statins alone or in combination with ezetimibe are the first line of treatment for HoFH patients, and other drugs are generally given in combination with statins. Since, HoFH is a severe form of Familial Hypercholesterolemia (FH), due to which LDL-C levels in the body are very high, patients are given high-dose statins to lower LDL-C levels. 

Three modalities are employed for treating HoFH to prevent heart disease by lowering cholesterol: lipoprotein (Lp) apheresis (LA), pharmacotherapy, and liver transplantation. Additionally, prescription medications can be used either alone or in combination with other treatments and medications. Furthermore, Juxtapid (lomitapide) and Repatha (evolocumab) are the US FDA-approved for the treatment of HoFH.

Even after the availability of so various therapeutic options, HoFH is highly underdiagnosed and undertreated. The expected launch of potential therapies such as LIB003 (LIB Therapeutics), Inclisiran (Novartis), and ARO-ANG3 (Arrowhead Pharmaceuticals) may increase the HoFH market size in the coming years. 

Inclisiran by Novartis is a long-acting, synthetic siRNA directed against PCSK9 and has been displayed to tremendously decrease hepatic production of PCSK9 and cause a marked decrease in LDL-C levels. It is conjugated to triantennary N-acetylgalactosamine carbohydrates administered via subcutaneous injection. In January 2018, Inclisiran was granted orphan drug designation by the US FDA for treatment of HoFH. 

ARO-ANG3 is designed to decrease the production of angiopoietin-like protein 3 (ANGPTL3), a liver synthesized inhibitor of lipoprotein lipase and endothelial lipase by Arrowhead Pharmaceuticals. ANGPTL3 inhibition has been displayed to reduce serum LDL, serum, and liver triglyceride and has genetic validation as a novel target for cardiovascular diseases and  Familial Hypercholesterolemia (FH). The US FDA has granted Orphan Drug Designation to ARO-ANG3 for the treatment of HoFH.

LIB003 is a PCSK9 Inhibitor being developed by LIB Therapeutics. The PCSK9 protein is a vital regulator of circulating LDL–C levels through its inhibitory action to recycle the LDL receptor (LDLR). PCSK9 inhibition hence offers a novel therapeutic mechanism for decreasing LDL–C levels. 

To conclude, the Homozygous Familial Hypercholesterolemia market dynamics are anticipated to change in the coming years due to the raising awareness of the disease, understanding the importance of cascade screening in several countries, initiation of comprehensive multicenter studies, improvement in the diagnosis methodologies, and incremental healthcare spending across the world during the forecast period. Currently, Evinacumab, Praluent, Inclisiran, and LIB003 can be expected to be the major thriving players in the HoFH market due to their highly efficacious results and the fact that they will enter the market much earlier than other HoFH therapies. Out of these therapies, Evinacumab has already received approval based on its brilliant results. On the other hand, LIB003's dosing advantage provides an edge to the drug over other PCSK9 monoclonal antibodies already available in the market, as it can demonstrate greater efficacy in a smaller injection volume as compared to PCSK9 monoclonal antibodies, and it has to be dosed only once a month. The formulation of LIB003 (LIB Therapeutics) provides an edge to the drug over other PCSK9 monoclonal antibodies already available in the HoFH market, as it can demonstrate greater efficacy in a smaller injection volume as compared to PCSK9 monoclonal antibodies. 

Scope of the Homozygous Familial Hypercholesterolemia Market Insight Report 

  • Geography Covered: The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan. 
  • Study Period: 3-year historical and 11-year forecasted analysis (2017-2030).
  • Homozygous Familial Hypercholesterolemia Markets Segmentation: By Geographies and By HoFH Therapies (Historical and Forecasted, Current and Upcoming) 
  • Dominant Market Companies investigating its candidates for HoFH: Amgen, Aegerion Pharmaceutical, Regeneron Pharmaceuticals, Alnylam Pharmaceuticals, Novartis, Lib Therapeutics, Sanofi, Neurobo Pharmaceuticals, Arrowhead Pharmaceuticals, and several others. 
  • Analysis: Comparative and conjoint analysis of emerging therapies. 
  • Case Studies
  • KOL's Views
  • Analyst's View

Request for a Webex demo of the report @ Homozygous Familial Hypercholesterolemia Therapeutics Market

Table of Contents 

1

Homozygous Familial Hypercholesterolemia (HoFH) Key Insights 

2

Homozygous Familial Hypercholesterolemia Executive Summary

3

Homozygous Familial Hypercholesterolemia Market Overview at a Glance

4

Homozygous Familial Hypercholesterolemia Disease Background And Overview

5

Recognized Establishments

6

Homozygous Familial Hypercholesterolemia Treatment And Management

7

HoFH Guidelines By European Atherosclerosis Society (EAS) (2014)

8

Japan Atherosclerosis Society (JAS) Guidelines For Prevention of Atherosclerotic Cardiovascular Diseases 2017

9

Guidance For Pediatric Familial Hypercholesterolemia (2017)

10

HoFH Treatment Algorithm

11

Homozygous Familial Hypercholesterolemia Epidemiology And Patient Population

11.1

The United States

11.2

EU5 Countries

11.2.1

Germany

11.2.2

France

11.2.3

Italy

11.2.4

Spain

11.2.5

The United Kingdom

11.3

Japan

12

Unmet Needs

13

HoFH Marketed Drugs

13.1

Repatha (Evolocumab/Amg 145): Amgen

13.2

Juxtapid (Lomitapide): Aegerion Pharmaceutical

13.3

Evkeeza (Evinacumab): Regeneron Pharmaceuticals

13.4

Praluent (Alirocumab): Sanofi/Regeneron Pharmaceuticals

14

Homozygous Familial Hypercholesterolemia Emerging Drugs

14.1

Inclisiran: Alnylam Pharmaceuticals/Novartis

14.2

Lib003: Lib Therapeutics

14.3

Gemcabene: Neurobo Pharmaceuticals

14.4

Aro-Ang3: Arrowhead Pharmaceuticals

15

HoFH Market Access And Reimbursement 

16

7MM Homozygous Familial Hypercholesterolemia Market Analysis

16.1

The United States Homozygous Familial Hypercholesterolemia Market Size

16.2

EU-5 Homozygous Familial Hypercholesterolemia Market Size

16.2.1

Germany Market Size

16.2.2

France Market Size

16.2.3

Italy Market Size

16.2.4

Spain Market Size

16.2.5

The United Kingdom Market Size

16.3

Japan Homozygous Familial Hypercholesterolemia Market Size

17

Case Reports of HoFH

18

Homozygous Familial Hypercholesterolemia Market Drivers

19

Homozygous Familial Hypercholesterolemia Market Barriers

20

SWOT Analysis Of Homozygous Familial Hypercholesterolemia

21

Appendix

22

DelveInsight Capabilities

23

Disclaimer

24

About DelveInsight

Browse full report with detailed TOC with charts, figures, tables @ Homozygous Familial Hypercholesterolemia Diagnostics Market Report

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