Hutchinson-Gilford Progeria Syndrome Market

Hutchinson-Gilford Progeria Syndrome Market

Global Hutchinson-Gilford progeria syndrome market reached US$ YY million in 2023 and is expected to reach US$ YY million by 2031, growing at a CAGR of YY% during the forecast period 2024-2031.

Hutchinson-Gilford progeria syndrome (HGPS) also known as Progeria, is a rare, fatal, genetic condition of childhood with striking features resembling premature aging. Children with progeria usually have a normal appearance in early infancy. At approximately nine to 24 months of age, affected children begin to experience profound growth delays, resulting in short stature and low weight. Children with progeria die of heart disease (atherosclerosis) at an average age of 14.5 years. As with any person suffering from heart disease, children with progeria can experience high blood pressure, strokes, angina, enlarged heart and heart failure, all conditions associated with aging.

Lonafarnib is an approved medication for HGPS, benefits include improved low-tone hearing, decreased headaches, decreased carotid-femoral pulse wave velocity, decreased arterial wall stiffness and increased life span. A regular diet with frequent small meals is recommended. Primary tooth extractions after the secondary tooth has erupted and/or fully descended may be required to avoid dental crowding. The use of sunscreen on all exposed areas of skin, including the head, is recommended for outdoor activities. Low-dose aspirin (2-3 mg/kg body weight) is recommended for the prevention of cardiovascular and stroke complications. Because the stiffened peripheral vasculature may be less tolerant to dehydration, maintaining optimal hydration orally is recommended.

Market Dynamics: Drivers

Rising awareness about the condition

The rising awareness about the condition and its associated complications is expected to drive the market over the forecast period. Improved awareness may lead to an earlier diagnosis of HGPS. Early identification of the condition is crucial for implementing interventions and treatments at a stage when they might be more effective in managing symptoms and improving the quality of life for affected individuals.

In addition, the increased awareness often correlates with greater public and private interest in supporting research initiatives. As more people become aware of HGPS, there may be an increase in funding for research and development of new therapies. Awareness campaigns can foster a sense of community and support for individuals and families affected by HGPS. Patient advocacy groups may play a crucial role in raising awareness, providing resources and advocating for better treatment options.

For instance, Rare Disease Day is February 28th and raises awareness for over 7,000+ rare diseases that impact 300 million people across the globe. By celebrating this day, the community hopes to advocate for rare disease research as well as gather support from the community for those impacted including Hutchinson-Gilford progeria syndrome, which is characterized by the accelerated appearance of premature aging starting in childhood. This disease affects one in 8 million children, with fewer than 100 cases documented in medical history.

Further, the increasing prevalence of Hutchinson-Gilford progeria syndrome, rising FDA approvals for novel therapies, increasing clinical trials and research activities to develop more advanced therapies and increasing advancements in the development of novel therapies are the factors expected to drive the market over the forecast period.

Restraints

Factors such as complications and side effects associated with the various therapies, the high cost of the treatment, availability of limited treatment options and lack of better and effective treatment, unmet needs and uncertainties regarding treatment response are the factors expected to hamper the market.

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Segment Analysis

The global Hutchinson-Gilford progeria syndrome market is segmented based on treatment type, end-user and region.

The farnesyltransferase inhibitors segment accounted for approximately 56.7% of the Hutchinson-Gilford progeria syndrome market share

The Farnesyltransferase Inhibitors (Zokinvy (Lonafarnib)) segment is expected to hold the largest market share over the forecast period. Lonafarnib is classified as a farnesyltransferase inhibitor. It works by inhibiting the farnesylation of progerin, a mutated protein associated with HGPS. Farnesylation is a post-translational modification process crucial for the functioning of certain proteins.

For instance, on November 20, 2020, Eiger BioPharmaceuticals, Inc cleared the U.S. Food and Drug Administration (FDA) approval of ZokinvyTM (lonafarnib) for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and processing-deficient Progeroid Laminopathies (PL).

Zokinvy, an FDA-approved farnesyltransferase inhibitor, is an oral medication that helps prevent the buildup of defective progerin or progerin-like protein. The effectiveness of Zokinvy for the treatment of Hutchinson-Gilford progeria syndrome was demonstrated in 62 patients from two single-arm trials that were compared to matched, untreated patients from a separate natural history study. Compared to untreated patients, the lifespan of Hutchinson-Gilford progeria syndrome patients treated with Zokinvy increased by an average of three months through the first three years of treatment and by an average of 2.5 years through the maximum follow-up time of 11 years.

Geographical Analysis

North America accounted for approximately 40.5% of the market share

North America region is expected to hold the largest market share over the forecast period owing to the increasing prevalence of the Hutchinson-Gilford progeria syndrome and increasing research activities in the region. North America, especially the United States is a hub to numerous research and development centers. These entities often play a pivotal role in advancing the understanding of rare diseases like Hutchinson-Gilford progeria syndrome and developing novel therapies.

In addition, there is an increasing prevalence of Hutchinson-Gilford progeria syndrome in the region, which increases the demand for novel therapeutics. For instance, according to the National Institute of Health, in the United States, HGPS occurs in one in four to eight million births and causes death at an average age of 14.5 years due to myocardial infarction or stroke.

Competitive Landscape

The major global players in the Hutchinson-Gilford progeria syndrome market include Eiger BioPharmaceuticals and among others.

COVID-19 Impact Analysis

The COVID-19 pandemic significantly impacted the global Hutchinson-Gilford progeria syndrome market. The coronavirus disease 2019 (COVID-19) pandemic led to disruptions in ongoing clinical trials across various therapeutic areas. Trials may have been delayed, suspended, or modified, impacting the development timeline for potential treatments for HGPS. Restrictions, lockdowns and changes in healthcare priorities during the pandemic could have affected access to medical care for individuals with rare diseases. This might include delayed diagnosis, treatment initiation, or access to specialized care.

Market Segmentation

By Treatment Type
• Farnesyltransferase Inhibitors (Zokinvy (Lonafarnib))
• Low-Dose Aspirin
• Physical and Occupational Therapy
• Hearing Aids
• Genetic Counseling
• Others

By End-User
• Hospitals
• Specialty Clinics
• Academic and Research Institutes
• Others

By Region
• North America
U.S.
Canada
Mexico
• Europe
Germany
U.K.
France
Spain
Italy
Rest of Europe
• South America
Brazil
Argentina
Rest of South America
• Asia-Pacific
China
India
Japan
Australia
Rest of Asia-Pacific
• Middle East and Africa

Why Purchase the Report?
• To visualize the global Hutchinson-Gilford progeria syndrome market segmentation based on treatment type, end-user and region, as well as understand key commercial assets and players.
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The global Hutchinson-Gilford progeria syndrome market report would provide approximately 26 tables, 42 figures and 187 Pages.

Target Audience 2023
• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies