Homozygous Familial Hypercholesterolemia Treatment Market: By Treatment Type (Statins, PCSK9 Inhibitors, Lomitapide, Mipomersen, LDL Apheresis, Gene Therapy), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, Online Pharmacies), and Geography

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.

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.