Lipodystrophy - Market Insight, Epidemiology, and Market Forecast - 2034

Key Highlights:

• Lipodystrophy is a rare disorder characterized by the abnormal distribution of body fat. It can result in the loss of fat tissue (lipoatrophy) or the accumulation of fat in abnormal areas of the body (lipohypertrophy).
• According to DelveInsight's estimates for 2023, there were approximately 3,077 prevalent cases of rare disease lipodystrophy across the 7MM, with the United States accounting for 35% of these cases.
• In 2023, 93% of diagnosed lipodystrophy cases were partial lipodystrophy, predominantly affecting females. This is largely because familial partial lipodystrophy (FPL), a common type, is linked to genetic mutations in the LMNA or PPARG genes, which are more prevalent in women.
• In 2023, approximately 30% of lipodystrophy cases in the United States were classified as congenital generalized lipodystrophy. This condition involves near-total loss of body fat, resulting in prominent muscles, severe insulin resistance, early-onset diabetes, hypertriglyceridemia, and non-alcoholic fatty liver disease (NAFLD).
• The US FDA has approved two therapies for Lipodystrophy, including MYALEPT (metreleptin) and EGRIFTA/EGRIFTA SV (tesamorelin) by Amryt Pharma and Thera Technologies, respectively.
• The pipeline for Lipodystrophy remains relatively limited, with only a few companies advancing their candidates through different stages of clinical trials. Notable candidates include REGN4461 by Regeneron Pharmaceuticals, which is currently in Phase II. The rarity of Lipodystrophy presents challenges in patient recruitment for clinical trials, which in turn slows the progress of developing new treatment options.
• The US represents the largest share of the lipodystrophy market, driven by the high prevalence of the condition and the availability of approved therapies. MYALEPT (metreleptin) by Amryt Pharma is a key player, with its leptin-based treatment generating significant revenue.

DelveInsight's "Lipodystrophy - Market Insights, Epidemiology and Market Forecast - 2034" report delivers an in-depth understanding of the indication Lipodystrophy, historical and forecasted epidemiology as well as the Lipodystrophy market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.

The Lipodystrophy market report provides real-world prescription pattern analysis, approved drugs, market share of individual therapies, and historical and forecasted 7MM Lipodystrophy market size from 2020 to 2034. The report also covers current Lipodystrophy treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's underlying potential.

Geography Covered:

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Lipodystrophy Understanding and Treatment Algorithm

Lipodystrophy Overview, Country-Specific Treatment Guidelines and Diagnosis

Lipodystrophy is a rare condition marked by abnormal fat distribution, either as fat loss (lipoatrophy) or fat accumulation (lipohypertrophy). It can be congenital (inherited) or acquired later in life. The disorder often leads to significant metabolic complications. Congenital lipodystrophy includes types like congenital generalized lipodystrophy (CGL), which involves widespread fat loss and metabolic issues like insulin resistance, and familial partial lipodystrophy (FPL), where fat is lost in some areas and accumulates in others. Acquired lipodystrophy can develop later, sometimes related to autoimmune diseases. Acquired generalized lipodystrophy (AGL) causes widespread fat loss, while acquired partial lipodystrophy (APL) leads to progressive upper body fat loss with lower body accumulation. HIV-associated lipodystrophy can occur as a side effect of antiretroviral therapy.

Symptoms include fat loss in the face, arms, legs, and buttocks, abnormal fat accumulation in the neck and trunk, and metabolic issues like insulin resistance and diabetes.

Diagnosis involves clinical evaluation, metabolic assessment, imaging studies like MRI or CT scans, and, in some cases, genetic testing or fat biopsy.

Lipodystrophy Treatment

Treatment for lipodystrophy primarily focuses on managing the metabolic complications that result from abnormal fat distribution, such as insulin resistance, diabetes, and hypertriglyceridemia. The approach is personalized based on the type of lipodystrophy and the severity of symptoms. Metabolic management includes medications like metformin for insulin resistance and insulin therapy for diabetes, along with drugs such as fibrates or statins to lower triglyceride levels and reduce cardiovascular risk. Lifestyle modifications involving diet and exercise are also essential for controlling blood sugar levels and maintaining overall health.

Lipodystrophy Epidemiology

The Lipodystrophy epidemiology chapter in the report provides historical as well as forecasted prevalence in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan from 2024 to 2034. The Lipodystrophy epidemiology is segmented with detailed insights into:

• Total cases of Lipodystrophy in the 7MM [2020-2034].
• Subtype-specific Cases of Lipodystrophy in the 7MM [2020-2034]
• Gender-specific Cases of Lipodystrophy in the 7MM [2020-2034]

Key Epidemiological Highlights

• According to DelveInsight's estimates for 2023, there were approximately 3,077 prevalent cases of rare disease lipodystrophy across the 7MM, with the United States accounting for 35% of these cases.
• In 2023, the EU4 countries and the UK together accounted for approximately 60% of the total lipodystrophy cases.
• In 2023, approximately 30% of lipodystrophy cases in the United States were classified as congenital generalized lipodystrophy. This condition involves near-total loss of body fat, resulting in prominent muscles, severe insulin resistance, early-onset diabetes, hypertriglyceridemia, and non-alcoholic fatty liver disease (NAFLD).
• In 2023, 93% of diagnosed lipodystrophy cases were partial lipodystrophy, predominantly affecting females. This is largely because familial partial lipodystrophy (FPL), a common type, is linked to genetic mutations in the LMNA or PPARG genes, which are more prevalent in women.

Lipodystrophy Drug Chapters

The drug chapter segment of the Lipodystrophy report encloses a detailed analysis of Lipodystrophy emerging and approved candidates. It also deep dives into the Lipodystrophy pivotal clinical trial details, recent and expected market approvals, patent details, the latest news, and recent deals and collaborations.

Approved Drugs

MYALEPT (metreleptin): Amryt Pharma

Metreleptin mimics the physiological effects of leptin by binding to and activating the human leptin receptor, which belongs to the Class I cytokine family of receptors that signals through the JAK/STAT transduction pathway. Only the metabolic effects of metreleptin have been studied. No effects on the distribution of subcutaneous fat are expected.

The US Food and Drug Administration (FDA) approved MYALEPT (metreleptin) in 2014, Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy patients: with confirmed congenital generalized lipodystrophy or acquired generalized lipodystrophy in adults and children 2 years of age and above.

EGRIFTA/EGRIFTA SV (tesamorelin): Thera Technologies

EGRIFTA/EGRIFTA SV is a growth hormone-releasing factor (GRF) analog indicated for reducing excess abdominal fat in HIV-infected patients with lipodystrophy. EGRIFTA is used to reduce lipodystrophy caused by certain HIV medications. EGRIFTA is not a weight-loss medication and should not be used to treat obesity.

The US Food and Drug Administration (FDA) approved EGRIFTA in 2010.

Emerging Drugs

REGN4461: Regeneron Pharmaceuticals

Mibavademab (REGN 4461) is a leptin receptor (LEPR) agonist antibody developed by Regeneron pharmaceuticals to treat generalized lipodystrophy. The drug is currently in Phase II clinical trials conducted on patients with generalized lipodystrophy and familial partial lipodystrophy.

Subcutaneous Metreleptin: Amryt Pharma

A 12-month randomized, multicenter, double-blind, placebo-controlled Phase III (METRE-PL) study to evaluate the safety and efficacy of daily subcutaneous metreleptin treatment in subjects with partial lipodystrophy.

Lipodystrophy Market Outlook

The market for lipodystrophy treatments is evolving with a focus on both established and emerging therapies. In the United States, the market is dominated by key approved therapies like MYALEPT (metreleptin) and EGRIFTA/EGRIFTA SV (tesamorelin), with substantial revenue contributions from these treatments.

• The US represents the largest share of the lipodystrophy market, driven by the high prevalence of the condition and the availability of approved therapies. MYALEPT (metreleptin) by Amryt Pharma is a key player, with its leptin-based treatment generating significant revenue, approximately $880,000.
• Among the EU4 countries and the UK, Germany has the largest market size for lipodystrophy treatments, valued at approximately USD 94 million. France follows, while the other EU4 countries and the UK have smaller market shares.
• REGN4461, currently in Phase II trials by Regeneron Pharmaceuticals, represents a promising emerging therapy for lipodystrophy. This investigational treatment, designed as a leptin receptor agonist, has the potential to address significant unmet needs in managing lipodystrophy by restoring metabolic balance and improving insulin sensitivity.

Drug Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2024-2034, which depends on the competitive landscape, safety, and efficacy data along with order of entry. It is important to understand that the key players evaluating their novel therapies in the pivotal and confirmatory trials should remain vigilant when selecting appropriate comparators to stand the greatest chance of a positive opinion from regulatory bodies, leading to approval, smooth launch, and rapid uptake.

Lipodystrophy Activities

This section provides insights into different therapeutic candidates. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

This section covers information on collaborations, acquisitions and mergers, licensing, and patent details for emerging therapies.

KOL Views

To keep up with the real-world scenario in current market trends, we take opinions from Key Industry leaders working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts were contacted for insights on the evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake along with challenges related to accessibility.

DelveInsight's analysts connected with 10+ KOLs to gather insights; however, interviews were conducted with 5+ KOLs in the 7MM. Their opinion helps understand and validate current treatment patterns of Lipodystrophy. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of gaps in disease diagnosis, patient awareness, physician acceptability, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided.

Conjoint Analysis analyzes multiple approved therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

In efficacy, the trial's primary and secondary outcome measures are evaluated; for instance, in event-free survival, one of the most important primary outcome measures is event-free survival and overall survival.

Further, the therapies' safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the probability of success, and the addressable patient pool for each therapy.

Market Access and Reimbursement

The section provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of currently used therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Scope of the Report:

• The report covers a segment of key events, an executive summary, descriptive overview of Lipodystrophy, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, and disease progression along with country specific treatment guidelines.
• Additionally, an all-inclusive account of the current therapies, along with the elaborative profiles of late-stage and prominent therapies, will have an impact on the current treatment landscape.
• A detailed review of the Lipodystrophy market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies, by understanding trends, through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help in shaping and driving the 7MM Lipodystrophy.

Lipodystrophy Report Insights

• Patient Population
• Therapeutic Approaches
• Lipodystrophy Pipeline Analysis
• Lipodystrophy Market Size and Trends
• Existing and future Market Opportunity

Lipodystrophy Report Key Strengths

• Eleven Years Forecast
• 7MM Coverage
• Lipodystrophy Epidemiology Segmentation
• Inclusion of Country specific treatment guidelines
• KOL's feedback on approved therapies
• Key Cross Competition
• Conjoint analysis
• Drugs Uptake and Key Market Forecast Assumptions

Lipodystrophy Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)

FAQs:

• What is the growth rate of the 7MM Lipodystrophy treatment market?
• What was the Lipodystrophy total market size, the market size by therapies, market share (%) distribution in 2020, and what would it look like in 2034? What are the contributing factors/key catalysts for this growth?
• Is there any unexplored patient setting that can open the window for growth in the future?
• What are the pricing variations among different geographies for approved and off-label therapies?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?
• What are the current options for the treatment of Lipodystrophy?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitations of existing therapies?
• Patient acceptability in terms of preferred treatment options as per real-world scenarios?
• What are the country-specific accessibility issues of expensive, recently approved therapies?

Reasons to buy:

• The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the Lipodystrophy Market.
• Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
• Detailed analysis and ranking of class-wise potential current therapies under the conjoint analysis section to provide visibility around leading classes.
• Highlights of access and reimbursement policies of approved therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.
• To understand Key Opinion Leaders' perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.