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Global Homozygous Familial Hypercholesterolemia Market Insight, Epidemiology and Market Forecast 2021-2030 -

DUBLIN--(BUSINESS WIRE)--The "Homozygous Familial Hypercholesterolemia - Market Insight, Epidemiology and Market Forecast - 2030" drug pipelines has been added to's offering.

This 'Homozygous Familial Hypercholesterolemia (HoFH) - Market Insights, Epidemiology and Market Forecast- 2030' report delivers an in-depth understanding of the HoFH , historical and forecasted epidemiology as well as the HoFH market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

Homozygous Familial Hypercholesterolemia (HoFH) Treatment

Given the severity of hypercholesterolemia with increased CV risk, HoFH requires intensive therapy. However, HoFH is often unresponsive to traditional treatment. The foundation of treatment for HoFH is an aggressive lowering of LDL-C levels in an attempt to bring levels to goal. This is often difficult to achieve despite multidrug treatment, given the markedly elevated levels of cholesterol at baseline and the limited response to available treatments. Statin treatment has been shown to affect CVD mortality in HoFH and statins are typically first-line treatment started at the time of diagnosis, including in very young children.

In HoFH, to prevent the incidence and progression of CAD, intensive lipid-lowering therapy should be initiated as early as possible at an early age. The major mechanisms of action of statins, bile acid adsorbing resins and PCSK9 inhibitors are to enhance expression (activation) of LDL receptors. For the defective type, in which only a small amount of LDL receptor activity remains, slight efficacy is observed, but in the negative type in which LDL receptor activity is completely absent, no LDL-C lowering effect is observed. However, as the frequencies of the adverse events of fatty liver and diarrhea are high, it is essential to control the fat and alcohol intake strictly. Probucol reportedly exerts a certain LDL-C lowering effect on HoFH and may cause the regression or disappearance of xanthoma in the skin or Achilles tendon). Nevertheless, for LDL-C control, LDL apheresis therapy once every 1-2 weeks is still required in many cases. When patients are resistant to all of the above treatments or show intolerance, liver transplantation may be considered.

Furthermore, in patients with HoFH, it is difficult to decrease the LDL-C level sufficiently using existing drug therapies, and many patients require continued LDL apheresis with extracorporeal circulation starting in childhood. Considering the inhibition of the progression of CAD, the earlier LDL apheresis is initiated, the better; however, it is difficult to perform LDL apheresis until the affected child can be kept in bed during apheresis.

Homozygous Familial Hypercholesterolemia (HoFH) Epidemiology

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Prevalence of Homozygous Familial Hypercholesterolemia (HoFH), Diagnosed Prevalence of Homozygous Familial Hypercholesterolemia (HoFH) and Mutation-specific Distribution of Homozygous Familial Hypercholesterolemia (HoFH) in the 7MM market covering the United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom) and Japan from 2017 to 2030.

Homozygous Familial Hypercholesterolemia (HoFH) Drug Chapters

The drug chapter segment of the Homozygous Familial Hypercholesterolemia (HoFH) report encloses the detailed analysis of Homozygous Familial Hypercholesterolemia (HoFH) marketed drugs and mid and late stage pipeline drugs. It also helps to understand the Homozygous Familial Hypercholesterolemia (HoFH) clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details of each included drug and the latest news and press releases.

Homozygous Familial Hypercholesterolemia (HoFH): Market Outlook

According to the FH Foundation, Homozygous Familial Hypercholesterolemia (HoFH) is a rare but serious autosomal dominant genetic condition that can lead to aggressive and premature heart disease, heart attacks, and strokes even in patients below 18 years. The indication has an estimated prevalence of 1 in 160,000 to 1 in 300,000 individuals.

The current market of HoFH comprises of several classes of treatment options, namely, Statins, MTP inhibitors (Lomitapide), PCSK9 inhibitors, and others (bile-acid-binding resins). In the case of advanced treatment options, Lipoprotein apheresis can also be opted to treat the patients with HoFH. While in rare and severe cases, liver transplantation can also be advised.

Out of these therapies, statins remain the first-line therapy for LDL-C lowering in HoFH, including in very young children, whereas, lomitapide remains the most powerful drug approved for HoFH with studies showing good response and tolerability for lomitapide. Common statin drugs that are usually prescribed for treating HoFH patients include Lipitor, Lescol, Pravachol, Crestor, Zocor, and others. Furthermore, statin treatment has been shown to affect CVD mortality in patients suffering from HoFH. Though this therapy is usually prescribed in combination with ezetimibe; however, this combination is far from adequate in achieving the required goal LDL-C levels in HoFH patients.

Since HoFH patients face a much more intensive challenge in managing their condition than an average person with high cholesterol. Therefore, to manage this condition, currently, there are two approved therapies available in the 7MM market, namely, Repatha (Amgen) and Juxtapid (Aegerion Pharmaceuticals).

Even after the availability of so many therapeutic options, HoFH is highly underdiagnosed and undertreated. This fact has also been highlighted by the European Atherosclerosis Society (EAS) Consensus Panel. Additionally, the organization also indicated that even at the highest doses of the most efficacious statins, only modest reductions in LDL C plasma levels, of 10-25%, are generally observed in HoFH patients. The EAS Consensus Panel suggests that the combination of statin plus ezetimibe can produce a further decrease in LDL-C levels of 10-15%. Furthermore, combination with other lipid-lowering treatments may also be considered, depending on their availability and tolerability.

Companies Mentioned

  • Amgen
  • Aegerion Pharmaceutical
  • Regeneron Pharmaceuticals
  • Alnylam Pharmaceuticals/Novartis
  • Lib Therapeutics
  • Sanofi/Regeneron Pharmaceuticals
  • Neurobo Pharmaceuticals
  • Arrowhead Pharmaceuticals

For more information about this drug pipelines report visit

Laura Wood, Senior Press Manager

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Last Updated: 01-Feb-2021