- Statins
- PCSK9 Inhibitors
- Lomitapide
- Mipomersen
- LDL Apheresis
- Gene Therapy
Homozygous Familial Hypercholesterolemia Treatment Market size was valued at USD 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.
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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.
The Homozygous Familial Hypercholesterolemia (HoFH) Treatment Market was valued at USD 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.
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 |