Homozygous Familial Hypercholesterolemia Treatment Market: By Treatment Type, By Distribution Channel, and Geography

Homozygous Familial Hypercholesterolemia Treatment Market Size, Share, Growth, Trends, and Global Industry Analysis: By Treatment Type (Statins, PCSK9 Inhibitors, Lomitapide, Mipomersen, LDL Apheresis, Gene Therapy), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, Online Pharmacies), and Region

Homozygous Familial Hypercholesterolemia Treatment Market size was valued at US$ 103.6 million in 2023 and is poised to grow at an 4.3% CAGR from 2024 to 2030. Homozygous Familial Hypercholesterolemia (HoFH) is a rare genetic disorder characterized by extremely high levels of low-density lipoprotein (LDL) cholesterol, leading to premature cardiovascular disease. The global homozygous familial hypercholesterolemia treatment market has been experiencing significant growth due to several key factors. The primary drivers include increased awareness among healthcare professionals and patients, improved diagnostic techniques, and the development of novel targeted therapies. The introduction of PCSK9 inhibitors, such as evolocumab and alirocumab, has revolutionized homozygous familial hypercholesterolemia treatment, offering more effective management of the disease and contributing substantially to market expansion.

Additionally, the approval of other targeted therapies like lomitapide and mipomersen has broadened treatment options for patients. The rarity of homozygous familial hypercholesterolemia, with an estimated prevalence of 1 in 160,000 to 1 in 300,000 individuals worldwide, presents opportunities for market growth. However, the homozygous familial hypercholesterolemia treatment market faces several challenges that act as restraints such as the high cost of novel therapies, particularly in developing countries, which limits access to treatment for many patients. The limited patient population due to the rarity of the disease can discourage pharmaceutical companies from investing in research and development. Furthermore, the complex nature of the disease and the need for lifelong treatment pose challenges in patient management and adherence. Despite these challenges, the homozygous familial hypercholesterolemia treatment market presents numerous opportunities. The potential for developing gene therapies and other advanced treatment modalities offers promising avenues for market expansion. Increasing collaborations between pharmaceutical companies, research institutions, and patient advocacy groups are likely to drive innovation and expand treatment options.

A recent development is the approval of Evkeeza (evinacumab-dgnb) by the U.S. Food and Drug Administration (FDA) on February 11, 2021. Developed by Regeneron Pharmaceuticals, Evkeeza is an angiopoietin-like 3 (ANGPTL3) inhibitor indicated as an adjunct to other LDL-lowering therapies for the treatment of homozygous familial hypercholesterolemia in patients 12 years and older. This approval represents a significant advancement in homozygous familial hypercholesterolemia treatment, offering a new mechanism of action to address the challenges of this rare genetic disorder. The great progression of biotechnologies enabled the rapid development of new therapeutic options, which are promising in gaining LDL-C targets in individuals with homozygous familial hypercholesterolemia. Besides the mentioned mAb and RNA-based therapeutics, gene transfer, silencing, and editing are currently under investigation for homozygous familial hypercholesterolemia.

Homozygous Familial Hypercholesterolemia Treatment Market Key Developments:
  • In July 2023, Novartis announced that the US Food and Drug Administration (FDA) has approved a label update for Leqvio (inclisiran) to enable earlier use in patients with elevated LDL-C who have an increased risk of heart disease, as an adjunct to diet and statin therapy.
  • In June 2023, Esperion announced that it had submitted Supplemental New Drug Applications (sNDA) to the U.S. Food and Drug Administration (FDA) for the Company's oral non-statin products NEXLETOL and NEXLIZET. In its sNDAs, Esperion seeks to add the use of both NEXLETOL and NEXLIZET for cardiovascular risk reduction and also seeks to remove the statin limitation in the LDL-C indication.

Homozygous Familial Hypercholesterolemia Treatment Market Summary

Study Period

2024-2030

Base Year

2023

CAGR

4.3%

Largest Market

North America

Fastest Growing Market

Asia Pacific

Homozygous Familial Hypercholesterolemia Treatment Market Dynamics

The global homozygous familial hypercholesterolemia treatment market is characterized by intense research activities aimed at developing more effective and convenient treatment options. Pharmaceutical companies are investing in the development of gene therapies, and RNA-based therapies, and exploring combination approaches to address the limitations of current treatment modalities. The increasing focus on personalized medicine and advancements in genetic testing techniques are expected to shape the future of homozygous familial hypercholesterolemia treatment. Moreover, patient advocacy groups and cardiovascular disease organizations play a crucial role in raising awareness about homozygous familial hypercholesterolemia, potentially leading to earlier diagnosis and treatment initiation. This, coupled with improving healthcare infrastructure in developing countries, is likely to expand the patient pool and drive market growth.

Homozygous Familial Hypercholesterolemia Treatment Market Segmentation Analysis

Report Benchmarks

Details

Report Study Period

2024-2030

Market Size in 2023

US$ 103.6 million

Market CAGR

4.3%

By Treatment Type

  • Statins
  • PCSK9 Inhibitors
  • Lomitapide
  • Mipomersen
  • LDL Apheresis
  • Gene Therapy

By Distribution Channel

  • Hospital Pharmacies
  • Retail Pharmacies
  • Online Pharmacies

Key Features of the Report

  • The report provides granular level information about the market size, regional market share, historic market (2019-2023), and forecast (2024-2030)
  • The report covers in-detail insights about the competitor’s overview, company share analysis, key market developments, and their key strategies
  • The report outlines drivers, restraints, unmet needs, and trends that are currently affecting the market
  • The report tracks recent innovations, key developments, and start-up details that are actively working in the market
  • The report provides a plethora of information about market entry strategies, regulatory framework, and reimbursement scenario

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Frequently Asked Questions

The Homozygous Familial Hypercholesterolemia (HoFH) Treatment Market was valued at US$ 103.6 million in 2023 and is expected to grow at an 4.3% CAGR from 2024 to 2030.

PCSK9 Inhibitors currently hold the highest market share in the homozygous familial hypercholesterolemia treatment market.

The Asia-Pacific region is estimated to grow at the highest CAGR over the forecast period.

The high cost of novel therapies limiting access to treatment, the limited patient population due to the rarity of the disease, and the complex nature of the disease and the need for lifelong treatment pose challenges in patient management and adherence, limiting the growth of the global homozygous familial hypercholesterolemia treatment market.

Author image

Author

Muni Kumar Meravath

Muni Kumar Meravath is a seasoned Healthcare Market Research Analyst with over 6 years of experience in the healthc.....

1.Executive Summary
2.Global Homozygous Familial Hypercholesterolemia Treatment Market Introduction 
2.1.Global Homozygous Familial Hypercholesterolemia Treatment Market  - Taxonomy
2.2.Global Homozygous Familial Hypercholesterolemia Treatment Market  - Definitions
2.2.1.Treatment Type
2.2.2.Treatment Type
2.2.3.Region
3.Global Homozygous Familial Hypercholesterolemia Treatment Market Dynamics
3.1. Drivers
3.2. Restraints
3.3. Opportunities/Unmet Needs of the Market
3.4. Trends
3.5. Product Landscape
3.6. New Product Launches
3.7. Impact of COVID 19 on Market
4.Global Homozygous Familial Hypercholesterolemia Treatment Market Analysis, 2019 - 2023 and Forecast 2024 - 2030
4.1.  Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
4.2.  Year-Over-Year (Y-o-Y) Growth Analysis (%) 
4.3.  Market Opportunity Analysis 
5.Global Homozygous Familial Hypercholesterolemia Treatment Market  By Treatment Type, 2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
5.1. Statins
5.1.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
5.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
5.1.3. Market Opportunity Analysis 
5.2. PCSK9 Inhibitors
5.2.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
5.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
5.2.3. Market Opportunity Analysis 
5.3. Lomitapide
5.3.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
5.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
5.3.3. Market Opportunity Analysis 
5.4. Mipomersen
5.4.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
5.4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
5.4.3. Market Opportunity Analysis 
5.5. LDL Apheresis
5.5.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
5.5.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
5.5.3. Market Opportunity Analysis 
5.6. Gene Therapy
5.6.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
5.6.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
5.6.3. Market Opportunity Analysis 
6.Global Homozygous Familial Hypercholesterolemia Treatment Market  By Treatment Type, 2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
6.1. Hospital Pharmacies
6.1.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
6.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
6.1.3. Market Opportunity Analysis 
6.2. Retail Pharmacies
6.2.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
6.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
6.2.3. Market Opportunity Analysis 
6.3. Online Pharmacies
6.3.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
6.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
6.3.3. Market Opportunity Analysis 
7.Global Homozygous Familial Hypercholesterolemia Treatment Market  By Region, 2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
7.1. North America
7.1.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
7.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
7.1.3. Market Opportunity Analysis 
7.2. Europe
7.2.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
7.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
7.2.3. Market Opportunity Analysis 
7.3. Asia Pacific (APAC)
7.3.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
7.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
7.3.3. Market Opportunity Analysis 
7.4. Middle East and Africa (MEA)
7.4.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
7.4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
7.4.3. Market Opportunity Analysis 
7.5. Latin America
7.5.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
7.5.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
7.5.3. Market Opportunity Analysis 
8.North America Homozygous Familial Hypercholesterolemia Treatment Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
8.1. Treatment Type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
8.1.1.Statins
8.1.2.PCSK9 Inhibitors
8.1.3.Lomitapide
8.1.4.Mipomersen
8.1.5.LDL Apheresis
8.1.6.Gene Therapy
8.2.  Treatment Type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
8.2.1.Hospital Pharmacies
8.2.2.Retail Pharmacies
8.2.3.Online Pharmacies
8.3.  Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
8.3.1.United States of America (USA)
8.3.2.Canada
9.Europe Homozygous Familial Hypercholesterolemia Treatment Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
9.1. Treatment Type Analysis  and Forecast  by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
9.1.1.Statins
9.1.2.PCSK9 Inhibitors
9.1.3.Lomitapide
9.1.4.Mipomersen
9.1.5.LDL Apheresis
9.1.6.Gene Therapy
9.2.  Treatment Type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
9.2.1.Hospital Pharmacies
9.2.2.Retail Pharmacies
9.2.3.Online Pharmacies
9.3.  Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
9.3.1.Germany
9.3.2.France
9.3.3.Italy
9.3.4.United Kingdom (UK)
9.3.5.Spain
10.Asia Pacific (APAC) Homozygous Familial Hypercholesterolemia Treatment Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
10.1. Treatment Type Analysis  and Forecast  by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
10.1.1.Statins
10.1.2.PCSK9 Inhibitors
10.1.3.Lomitapide
10.1.4.Mipomersen
10.1.5.LDL Apheresis
10.1.6.Gene Therapy
10.2.  Treatment Type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
10.2.1.Hospital Pharmacies
10.2.2.Retail Pharmacies
10.2.3.Online Pharmacies
10.3.  Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
10.3.1.China
10.3.2.India
10.3.3.Australia and New Zealand (ANZ)
10.3.4.Japan
10.3.5.Rest of APAC
11.Middle East and Africa (MEA) Homozygous Familial Hypercholesterolemia Treatment Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
11.1. Treatment Type Analysis  and Forecast  by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
11.1.1.Statins
11.1.2.PCSK9 Inhibitors
11.1.3.Lomitapide
11.1.4.Mipomersen
11.1.5.LDL Apheresis
11.1.6.Gene Therapy
11.2.  Treatment Type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
11.2.1.Hospital Pharmacies
11.2.2.Retail Pharmacies
11.2.3.Online Pharmacies
11.3.  Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
11.3.1.GCC Countries
11.3.2.South Africa
11.3.3.Rest of MEA
12.Latin America Homozygous Familial Hypercholesterolemia Treatment Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
12.1. Treatment Type Analysis  and Forecast  by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
12.1.1.Statins
12.1.2.PCSK9 Inhibitors
12.1.3.Lomitapide
12.1.4.Mipomersen
12.1.5.LDL Apheresis
12.1.6.Gene Therapy
12.2.  Treatment Type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
12.2.1.Hospital Pharmacies
12.2.2.Retail Pharmacies
12.2.3.Online Pharmacies
12.3.  Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
12.3.1.Brazil
12.3.2.Mexico
12.3.3.Rest of LA
13. Competition Landscape
13.1.  Market Player Profiles (Introduction, Brand/Product Sales, Financial Analysis, Product Offerings, Key Developments, Collaborations, M & A, Strategies, and SWOT Analysis) 
13.2.1.Amgen Inc.
13.2.2.Sanofi
13.2.3.Novartis AG
13.2.4.Regeneron Pharmaceuticals, Inc.
13.2.5.Ionis Pharmaceuticals, Inc.
13.2.6.Teva Pharmaceutical Industries Ltd.
13.2.7.Esperion Therapeutics, Inc.
13.2.8.AstraZeneca plc
13.2.9.Pfizer Inc.
13.2.10.Merck & Co., Inc.
14. Research Methodology 
15. Appendix and Abbreviations 

Key Market Players

  • Amgen Inc.
  • Sanofi
  • Novartis AG
  • Regeneron Pharmaceuticals, Inc.
  • Ionis Pharmaceuticals, Inc.
  • Teva Pharmaceutical Industries Ltd.
  • Esperion Therapeutics, Inc.
  • AstraZeneca plc
  • Pfizer Inc.
  • Merck & Co., Inc.

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