Familial Hypercholesterolemia Treatment Market Size, Share, Industry, Forecast and outlook (2024-2031)

Familial Hypercholesterolemia Treatment Market is Segmented By Drug Type (Statins, Combination cholesterol absorption inhibitor and statin, Ezetimibe (Zetia), PCSK9 inhibitors, Fibrates, Omega-3 fatty acid supplements, Others), By Indication Type (Heterozygous familial hypercholesterolemia, Homozygous familial hypercholesterolemia), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, Other), By Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) – Share, Size, Outlook, and Opportunity Analysis, 2024-2031

Report Overview

The Familial Hypercholesterolemia Treatment Market size was valued at US$ YY million in 2023 and is estimated to reach US$ YY million by 2031, growing at a high CAGR during the forecast period (2024-2031).

Familial hypercholesterolemia is a genetic disease that runs in families. It raises LDL (bad) cholesterol levels dramatically. The disease starts at birth and can lead to heart attacks at a young age.

Market Summary

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Market Dynamics

The factors influencing the familial hypercholesterolemia treatment market are the increasing FDA approvals and demand for familial hypercholesterolemia treatment and the growing prevalence of familial hypercholesterolemia.

The increasing FDA approvals and demand for familial hypercholesterolemia treatment are expected to drive the market growth

Familial hypercholesterolemia is a genetic disease that runs in families. It raises LDL (bad) cholesterol levels dramatically. The disease starts at birth and can lead to heart attacks at a young age. LDL cholesterol levels of over 190 mg/dL in adults (and over 160 mg/dL in children) are among the most common signs of FH. Furthermore, most FH people have a family history of early heart disease or heart attacks. Routine blood cholesterol screening can reveal elevated LDL levels in some cases.

The use of novel therapies as an adjunctive treatment for HoFH has been approved. Lomitapide, a microsomal triglyceride transfer protein inhibitor, can lower LDL-C by up to 46 percent in people over 18. In addition, injectable mipomersen, an antisense RNA therapy, is approved in the United States for children as young as 12 years old and can lower LDL-C by up to 25%. Monoclonal antibodies to PCSK9 are being touted as a new treatment option for FH. Moreover, the increasing FDA approvals are expected to drive market growth. For instance, on April 1st, 2021, Praluent (alirocumab) injection was approved by the Food and Drug Administration (FDA) for adult patients with homozygous familial hypercholesterolemia (HoFH). This genetic condition causes severely high cholesterol. Praluent isn't meant to be used on its own; it's meant to be combined with other HoFH treatments.

Limitations associated with familial hypercholesterolemia disease are expected to hamper the market growth

FH is frequently misdiagnosed and mistreated, especially in children. 10 Primary care providers bear the burden of early FH diagnosis. They have a unique opportunity to improve FH detection and management. 11 The National Heart, Lung, and Blood Institute, American Academy of Pediatrics, American Heart Association, National Lipid Association, and American College of Cardiology all recommend universal lipid screening in children aged 9 to 11 years, which has the potential to significantly improve case finding. Moreover, there are currently no long-term studies in children with FH that show a reduction in vascular events when LDL-C lowering treatment is started early. According to the study, children with FH have significantly greater carotid intimamedia thickness than their unaffected siblings, which can be detected as early as 8 to 10 years old.

COVID-19 Impact Analysis

Patients with familial hypercholesterolemia (FH) are more likely to develop COVID-19 complications during the infection's acute phase and for a long time afterward. Because the level of low-density lipoprotein cholesterol (LDL-C) in FH patients is elevated from birth and correlates with the degree of systemic endothelial dysfunction, both heterozygous FH (HeFH) and, in particular, homozygous FH (HoFH) patients have a dysfunctional endothelium that is vulnerable to further damage from the direct viral attack and the hyper-inflammatory response typical of severe COVID-19. The use of statins in patients with COVID-19 has been beneficial in the past. Therefore, the focus on FH patients should be on lowering LDL-C levels effectively, as this is the root cause of the expected increased vulnerability to COVID-19 infection. During the COVID pandemic, continued use of statins and other lipid-lowering therapies should be encouraged to reduce the risk of COVID-19-related cardiovascular complications. Hence, the covid-19 is expected to positively impact the market growth.

Market Segment Analysis

Based on drugs, the statins segment is expected to drive the market growth in the forecast period

Statins are microorganism-derived drugs that significantly reduce cholesterol synthesis in cells by inhibiting the enzyme HMG-CoA reductase's activity. Statins, on the other hand, lower cholesterol levels in the blood by increasing the number of high-affinity receptors that recognize plasma LDL and decreasing the rate of VLDL synthesis in the liver. This explains why these drugs have a minor triglyceride-lowering effect. The efficacy of statins in lowering plasma cholesterol levels is comparable to that of bile acid-binding polymers, but it is superior to that of fibrates and nicotinic acid. Statins have a better tolerability profile than the other two drugs, but they are less effective at lowering plasma triglycerides and increasing HDL cholesterol levels. At their maximum approved doses, the strongest statins, rosuvastatin, and atorvastatin, can reduce LDLc levels by 50-60%. Moreover, the FDA approvals of this drug are expected to boost its market growth. For instance, on 22nd December 2021, the FDA approved Leqvio (inclisiran) injection for adults with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (ASCVD) who require additional low-density lipoprotein lowering in addition to diet and maximally tolerated statin therapy.

Market Geographical Share

North America region is expected to hold the largest market share in the global familial hypercholesterolemia treatment market

The growing prevalence of familial hypercholesterolemia and increasing FDA approvals in this region is expected to drive the market growth.

The prevalence of probable/definite FH in the United States was estimated at 0.40 percent (95 percent confidence interval [CI], 0.32-0.48), or 1 in 250 people (95 percent CI, 1 in 311 to 209). This means that 834,500 adults in the United States have FH. According to studies, the prevalence of heterozygous FH in the general population in the United States and worldwide is 1 in 250.

Moreover, on 23rd December 2021, The US Food and Drug Administration (FDA) approved Inclisiran (Leqvio) for lowering LDL-C in patients with atherosclerotic cardiovascular disease or heterozygous hypercholesterolemia.

Market Competitive Landscape

The global Familial Hypercholesterolemia Treatment Market is moderately competitive with mergers, acquisitions, and product launches. Some of the key players in the market are Amryt Pharmaceuticals, Inc., Novartis AG, AstraZeneca Plc., Regeneron Pharmaceuticals, Sanofi S.A., Amgen Inc., Daiichi Sankyo Company Limited, Esperion Therapeutics, Viatris Inc., Kowa Pharmaceuticals America, Inc. and Merck & Co., Inc.

Novartis AG

Overview: Novartis International AG, based in Basel, Switzerland, is a multinational pharmaceutical corporation. It is one of the world's largest pharmaceutical corporations. The company was founded in 1996.

Product Portfolio: LEQVIO (inclisiran) is an injectable prescription medicine used in adults who need additional lowering of "bad" cholesterol (LDL-C) and have  heterozygous familial hypercholesterolemia (HeFH).

Key Development: On 23rd December 2021, The US Food and Drug Administration (FDA) approved Novarti’s Inclisiran (Leqvio) to lower LDL-C in patients with atherosclerotic cardiovascular disease heterozygous hypercholesterolemia.