Hutchinson-Gilford Syndrome Market

Market Overview:

The 7 major Hutchinson-Gilford syndrome markets are expected to exhibit a CAGR of 6.53% during 2024-2034.

The Hutchinson-Gilford Syndrome market has been comprehensively analyzed in IMARC's new report titled "Hutchinson-Gilford Syndrome Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Hutchinson-Gilford Syndrome, also known as progeria, is an extremely rare genetic condition that causes accelerated aging in children. It is caused by a spontaneous mutation in the LMNA gene, which results in the production of an abnormal protein called progerin. The symptoms of this illness typically appear in the first year of life and include growth failure, a distinctive appearance characterized by a small face and jaw, thinning hair, and loss of subcutaneous fat. Individuals suffering from the disorder may also experience stiffness in joints, wrinkled skin, high-pitched voices, veins easily seen through the skin, dental problems, premature aging, a weakened immune system, etc. The diagnosis of Hutchinson-Gilford Syndrome is mainly based on the patient's reported clinical features and medical history. The healthcare provider may also perform a physical test to measure the height, weight, and vital signs of patients. Additionally, genetic testing is required to confirm a diagnosis by determining changes in the gene associated with the underlying disease.

The increasing cases of gene abnormalities that cause an unstable cell nucleus structure, thereby affecting normal cell division, are primarily driving the Hutchinson-Gilford Syndrome market. Furthermore, the widespread adoption of farnesyltransferase inhibitors owing to their various advantages, such as increased flexibility of blood vessels, improved bone structure, and enhanced hearing capabilities in patients, is also augmenting the market growth. In addition to this, the escalating utilization of physical and occupational therapy, which eases disease symptoms and promotes a person's ability to perform daily tasks by maintaining a good range of motion, balance, and posture, is further creating a positive outlook for the market. Moreover, several key players are investing in R&D activities to understand better the molecular pathways involved in the pathogenesis of disease and help to launch more potent treatment alternatives. This, in turn, is also bolstering market growth. Additionally, the emerging popularity of antisense oligonucleotide therapies, since they can inactivate harmful genes and decrease the level of telomeric non-coding RNA, is expected to drive the Hutchinson-Gilford Syndrome market in the coming years.

IMARC Group's new report provides an exhaustive analysis of the Hutchinson-Gilford Syndrome market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report, the United States has the largest patient pool for Hutchinson-Gilford Syndrome and represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc. have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the Hutchinson-Gilford Syndrome market in any manner.

Recent Developments:

• In January 2024, Eiger BioPharmaceuticals, Inc., announced that the company and its partner AnGes, Inc. received marketing approval from the Ministry of Health, Labour and Welfare for Zokinvy (lonafarnib), a treatment for Hutchinson-Gilford Syndrome (HGPS).
• In October 2023, The Progeria Research Foundation unveiled a groundbreaking Progeria test that accelerates the assessment of treatment benefits, indicating extended lifespans with lonafarnib.
• In April 2023, the application of Progerinin in HGPS model mice demonstrated the ability to improve cardiac function and address arterial irregularities. These findings suggest promising prospects for Progerinin's effectiveness in treating cardiac dysfunction linked to HGPS.

Key Highlights:

• With a prevalence of 1 in 23 million, Hutchinson-Gilford Syndrome (HGPS) is primarily caused by LMNA mutations, accounting for 92.8% of cases, while the remainder are linked to ZMPSTE24 mutations. Skin irregularities typically emerge earliest in HGPS patients, serving as a crucial diagnostic indicator.
• The prevalence of children with HGPS per total population is one in 20 million.
• The projected rate of occurrence for Hutchinson-Gilford Syndrome (HGPS) at birth is one in four million, showing consistent prevalence regardless of ethnic origin.
• There are approximately 350-400 children globally who are estimated to have Progeria. However, only 161 cases have been identified by The Progeria Research Foundation, leaving around 200 children without a diagnosis.

Drugs:

Zokinvy (lonafarnib) represents the first FDA-approved treatment designed to reduce mortality risk in children diagnosed with Hutchinson-Gilford Syndrome. Acting as a farnesyltransferase inhibitor, it works to hinder the buildup of faulty, farnesylated proteins by blocking farnesyltransferase activity. Initially developed by Merck, lonafarnib was later licensed to Eiger Biopharmaceuticals Inc., which currently markets it under the Zokinvy brand. The FDA approved on November 20, 2020, marking a significant milestone as the first FDA-approved therapy for HGPS and related progeroid laminopathies. Additionally, it is indicated for the treatment of processing-deficient progeroid laminopathies in the same patient population, including individuals with either a heterozygous LMNA mutation leading to progerin-like protein accumulation or homozygous/compound heterozygous mutations in ZMPSTE24.

Time Period of the Study

• Base Year: 2023
• Historical Period: 2018-2023
• Market Forecast: 2024-2034
   

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan
   

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the Hutchinson-Gilford syndrome market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the Hutchinson-Gilford syndrome market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current Hutchinson-Gilford syndrome marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the Hutchinson-Gilford syndrome market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
• What was the country-wise size of the Hutchinson-Gilford syndrome market across the seven major markets in 2023 and what will it look like in 2034?
• What is the growth rate of the Hutchinson-Gilford syndrome market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?
   

Epidemiology Insights

• What is the number of prevalent cases (2018-2034) of Hutchinson-Gilford syndrome across the seven major markets?
• What is the number of prevalent cases (2018-2034) of Hutchinson-Gilford syndrome by age across the seven major markets?
• What is the number of prevalent cases (2018-2034) of Hutchinson-Gilford syndrome by gender across the seven major markets?
• How many patients are diagnosed (2018-2034) with Hutchinson-Gilford syndrome across the seven major markets?
• What is the size of the Hutchinson-Gilford syndrome patient pool (2018-2023) across the seven major markets?
• What would be the forecasted patient pool (2024-2034) across the seven major markets?
• What are the key factors driving the epidemiological trend of Hutchinson-Gilford syndrome?
• What will be the growth rate of patients across the seven major markets?
   

Hutchinson-Gilford Syndrome: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for Hutchinson-Gilford syndrome drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the Hutchinson-Gilford syndrome market?
• What are the key regulatory events related to the Hutchinson-Gilford syndrome market?
• What is the structure of clinical trial landscape by status related to the Hutchinson-Gilford syndrome market?
• What is the structure of clinical trial landscape by phase related to the Hutchinson-Gilford syndrome market?
• What is the structure of clinical trial landscape by route of administration related to the Hutchinson-Gilford syndrome market?