Global Homozygous Familial Hypercholesterolemia Market - 2023-2030

Overview

Global Homozygous Familial Hypercholesterolemia Market reached US$ 83.7 million in 2022 and is expected to reach US$ 101.8 million by 2030 growing with a CAGR of 2.5% during the forecast period 2023-2030. The trend such as the shift towards advancements in genetic diagnostics due to its precise potential in identification of genetic mutations responsible for HoFH, facilitating early diagnosis is expected to dominate the global homozygous familial hypercholesterolemia market.

The global homozygous familial hypercholesterolemia market has grown significantly in recent years and is projected to continue on its upward trend. The market is undergoing a transformational period, influenced by several significant trends such development of novel technologies like CRISPR-Cas9 and RNA interference (RNAi).

Furthermore, the rising prevalence of cardiovascular disorders, launch of potential emerging therapies such as monoclonal antibodies, rising awareness of the disease, increasing number of clinical trials are driving up the homozygous familial hypercholesterolemia market size.

The growing research activities for treating disease using homozygous familial hypercholesterolemia approach and presence of establish biopharmaceutical and pharmaceutical companies are some of the factors in demand from North American regions. With significant competitors like AstraZeneca, Viatris Inc., Accord Healthcare, and others actively operating in the market.

Dynamics

The Increasing Prevalence of Cardiovascular Disease to Drive the Growth of the Homozygous familial hypercholesterolemia Market

The rising prevalence of cardiovascular diseases (CVDs) is a major driver for the homozygous familial hypercholesterolemia (HoFH) market. As HoFH is characterized by high levels of LDL-C and increased risk of CVDs like heart attacks and strokes, growing awareness of this disease fuels early detection and interventions that in turn drive demand for efficient HoFH treatments.

For instance, according to Centers for Disease Control and Prevention 2023 report, heart disease disease is one of the leading cause of death in U.S. Every 40 seconds some or other person in U.S. suffer from heart attack. In U.S. every year, approximately 805,000 people have heart attack. Moreover coronary heart disease is the most common type of heart disease, killing 375,476 people in 2021. As the CVD and coronary heart disease burden continues to increase, the HoFH market is expected to grow during the forecast period.

The Growing Advancements in Gene Therapies will Drive the Homozygous Familial Hypercholesterolemia Market Growth

The growing technological advancement in homozygous familial hypercholesterolemia such as CRISPR-Cas9, small interfering RNA (SiRNA), exosome-mediated therapy and others is expected to drive the demand for the market. The CRISPR/Cas9 system is revolutionary third generation gene editing tool after ZFNs and TALENs and currently the genome editing tool of choice.

For instance, according to National Library of Medicine 2022 article state that, the adeno-associated virus (AAV) containing the CRISPR/Cas9 gene editing system (AAV-CRISPR/Cas9) was used in a recent in vivo animal study to target LDLR mutant mice with impaired LDL receptor function (AAV-CRISPR/Cas9). The researchers discovered that using AAV-CRISPR/Cas9 gene editing, they were able to partially fix the mutation in hepatocytes, restoring LDL receptor protein expression.

This resulted in major decreases in serum total cholesterol, triglyceride, and LDL cholesterol levels. As a result, atherosclerotic plaque formation in the aorta reduced significantly, indicating CRISPR/Cas9's potential promise for treating heterozygous and homozygous familial hypercholesterolemia. Thus, advancements in technologies for gene therapies will drive the demand for the market over the forecast period.

The High Cost Associated with the Homozygous Familial Hypercholesterolemia Treatment Will Hamper the Growth of the Market

The high cost of treatment for homozygous familial hypercholesterolemia is a significant barrier for this market. The high costs of these medications may make them difficult to afford for patients, preventing many people from obtaining these vital therapies. This financial burden can affect more persons in low-income areas, causing gaps in who can receive therapies that can save lives for patients with HoFH.

For instance, Lomitapide drug, sold under the brand of Juxtapid in U.S for reducing the low-density lipoprotein (LDL) apheresis cost around cost around. This oral 5 mg capsule cost around $53,471 for a supply of 28 capsules. Moreover, unavailability of generic version may find it difficult to afford for the pateint in developing and low income countries. Thus, owing to the above factors the market is expected to face a restraint over the foreseeable future.

Segment Analysis

The global homozygous familial hypercholesterolemia market is segmented based on drug class, route of administration, technology, distribution channel, and region.

Owing to the Better Effectiveness, the Statins Segment Accounted for Approximately 47.6% of the Homozygous Familial Hypercholesterolemia Market Share

The statins segment is poised to dominate the homozygous familial hypercholesterolemia market due to effectiveness in reducing LDL cholesterol levels and managing cardiovascular risk. Statins are the mainstay treatment for treating the homozygous familial hypercholesterolemia due to its ability to reduce the LDL-C by diminishing hepatic cholesterol synthesis acting on the 3-hydroxy-3-methylglutaryl coenzyme-A (HMG-CoA)-reductase.

Also, the rising prevalence of atherosclerosis is major factors where treatment such as statins are very effective. For instance, according to Family Heart Foundation studies, it has shown that statins can reduce LDL-cholesterol by 35 to 55% and can reduce the risk of cardiovascular disease by 22% for each 40 mg/dL reduction in LDL-cholesterol.

Currently, rosuvastatin has the most potent lipid-lowering capability of any presently available statin and is suggested for people with primary hyperlipidemia and mixed dyslipidemia, homozygous FH, primary dysbetalipoproteinemia, and hypertriglyceridemia. Also, statins are ery effective in children and are proven to be safe with children suffering from lipid disorders.

Moreover, statins are more effective when given in combination with other adjuncts treatment such as evinacumab, PCSK9 inhibitors, and other. Many new treatment are that are use as adjuncts to statins are expected to get FDA approval. Thus, due to statins better effectiveness the segment is expected to dominate over the forecast period.

Geographical Penetration

North America Accounted for Approximately 41.7% of the Market Share in 2022, Owing to the Rising Prevalence of Atherosclerotic Cardiovascular Disease (ASCVD)

North America, particularly the U.S., dominates the global homozygous familial hypercholesterolemia market due to presence of huge number of key players settled in the region, growing prevalence of atherosclerotic cardiovascular disease (ASCVD), and high research activities along with developments of technologies by major organization in the region.

For instance, according to National Librabry of Medicine 2022 article, approximately the prevalence of ASCVD in the US was about 24 million in 2019, approximately 10% of the total US population are above 21 years old. It was found heavy comorbidity burden among ASCVD patients and 31.2% were at very high risk for recurrent events. Thus, owing to above factors the region is expected to grow over the forecast period.

Competitive Landscape

The major global players in the homozygous familial hypercholesterolemia market include: AstraZeneca, Viatris Inc., Teva Pharmaceutical Industries Ltd., Accord Healthcare, Changzhou Pharmaceutical Factory, Regeneron Pharmaceuticals, Inc., Amryt Pharma plc, Amgen Inc., Organon, Global Inc., CMP Pharma, and among others.

COVID-19 Impact Analysis

The COVID-19 pandemic has had a significant impact on the lifestyle and management of people with FH, especially homozygous FH (HoFH). Fear of COVID-19 has caused many to avoid getting medical attention. Furthermore, because FH patients were designated high-risk groups due to their greater vulnerability to COVID-19 infection, FH patients' access to cardiovascular therapy and prevention during the pandemic was limited.

For intance, according to the National Librabry of Medicine 2022 article, this population-based study found that FH patients with COVID-19 have a considerably increased risk of acute myocardial infarction. Overall, the COVID-19 pandemic has established hurdles to cardiovascular therapy for patients with FH, particularly HoFH. Thereby creating a major impact on the homozygous familial hypercholesterolemia market.

By Drug Class

• Statins
• Cholesterol Absorption Inhibitors
• PCSK9 Inhibitors
• MTP Inhibitors
• ANGPTL3 Inhibitors

By Route of Administration

• Oral
• Parenteral
• Nasal

By Technology

• CRISPR-Cas9
• RNA Interference
• Nanoparticle-Based Therapies

By Distribution Channel

• Hospitals
• Retail Pharmacies
• Online Pharmacies

By Region

• North America
  • U.S.
  • Canada
  • Mexico

• Europe
  • Germany
  • U.K.
  • France
  • Spain
  • Italy
  • Rest of Europe

• South America
  • Brazil
  • Argentina
  • Rest of South America

• Asia-Pacific
  • China
  • India
  • Japan
  • Australia
  • Rest of Asia-Pacific

• Middle East and Africa

Key Developments

• On March 22, 2023, Regeneron Pharmaceuticals, Inc. announced that the FDA has extended the approval of Evkeeza (evinacumab-dgnb) as an addition to other lipid-lowering medicines in children aged 5 to 11 with homozygous familial hypercholesterolemia (HoFH). Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor medication approved for children as young as 5 years old to decrease dangerously high levels of low-density lipoprotein cholesterol (LDL-C) caused by HoFH.
• On April 1, 2021, The FDA has approved Praluent (alirocumab) injectable for adult patients with homozygous familial hypercholesterolemia (HoFH), a hereditary disease that causes significantly elevated cholesterol. Praluent is not intended to be taken alone, but rather to be combined with other HoFH therapies.

DMI Opinion

The Global Homozygous Familial Hypercholesterolemia (HoFH) Market is expected to rise rapidly, because of advances in genetic diagnostics, gene treatments such as CRISPR-Cas9, and growing awareness of the condition. The link between HoFH and cardiovascular disorders creates demand for effective treatments, but addressing treatment affordability and accessibility is essential. While gene editing technologies have immense possibilities, but don't come without problems and safety issues.

The market is segmented based on several characteristics, with North America dominant due to research capability and disease incidence. New treatments such as Evkeeza and Praluent demonstrate continued innovation, whereas COVID-19 highlights the importance of robust healthcare systems and equal access to therapy. Balancing developments with ethical issues remain crucial in navigating this landscape.

Why Purchase the Report?

• To visualize the global homozygous familial hypercholesterolemia market segmentation based on drug class, route of administration, technology, distribution channel and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development.
• Excel data sheet with numerous data points of homozygous familial hypercholesterolemia market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping is available in Excel consisting of key products of all the major players.

The global homozygous familial hypercholesterolemia market report would provide approximately 53 tables, 54 figures and 195 Pages.

Target Audience 2023

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies