Homocystinuria Market

 Market Overview:

The 7 major homocystinuria markets are expected to exhibit a CAGR of 3% during 2024-2034.

The homocystinuria market has been comprehensively analyzed in IMARC's new report titled "Homocystinuria Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Homocystinuria is a rare genetic disorder that mainly affects the ability of the body to break down certain amino acids, specifically methionine. The most common type of homocystinuria is caused by a deficiency of an enzyme called cystathionine beta-synthase (CBS). This can lead to excess formation of homocysteine and other related substances in the blood and urine. Symptoms of homocystinuria can vary widely and may include developmental delays, intellectual disability, seizures, visual problems, chest deformities, abnormal bone development, blood clot formation, etc. In some cases, patients may also experience failure to gain weight at an expected rate, movement problems, spindly arms and legs, dislocation of the lens at the front of the eye, seizures, nearsightedness, etc. The diagnosis of homocystinuria is typically based on the patient's medical history, indications, and physical exam. A blood test is also utilized to measure elevated levels of amino acids in the body. The healthcare provider may further perform various studies, including genetic testing, liver biopsies, hepatic enzyme tests, etc., to validate a disease diagnosis among patients.

The rising cases of genetic disorders, causing gene mutations that can prevent the normal functioning of the enzymes, are primarily driving the homocystinuria market. Besides this, the widespread adoption of vitamin B6 and folic acid supplements, which can decrease blood levels of homocysteine and provide symptom relief, is also augmenting the market growth. Furthermore, the escalating application of lifestyle modifications, such as consuming a low-methionine diet, avoiding alcohol, practicing aerobic and strength-training exercises, etc., to slow the disease progression and enhance the quality of life in patients, is creating a positive outlook for the market. Additionally, various key players are making significant investments in R&D activities to launch novel therapeutic formulations that can control elevated levels of amino acids in critical illnesses. This, in turn, is further bolstering the market growth. Moreover, the emerging popularity of PEGylated recombinant enzyme replacement therapy with pegtibatinase, which aims to introduce the CBS enzyme into circulation, thereby reducing homocysteine concentrations, is expected to drive the homocystinuria market in the coming years.

IMARC Group's new report provides an exhaustive analysis of the homocystinuria 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 homocystinuria and also 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 homocystinuria market in any manner.

Recent Developments:

• In February 2024, Synlogic, Inc. announced the publication of a manuscript describing the development and activity of SYNB1353 in preclinical models, as well as demonstrating safety, tolerability, and clinical evidence of mechanism in healthy volunteers by successfully lowering methionine (Met), a precursor to homocysteine, in a dietary model of classical homocystinuria (HCU).
• In December 2023, Travere Therapeutics, Inc. stated that recruitment has opened in the HARMONY Study, a global, randomized pivotal Phase 3 clinical trial of pegtibatinase, a new investigational enzyme replacement medication being assessed for the treatment of HCU.
• In April 2023, Aeglea BioTherapeutics, Inc. announced interim results from its Phase 1/2 clinical study of pegtarviliase, a new recombinant human enzyme intended to lower excessive levels of total homocysteine, for the treatment of HCU.

Key Highlights:

• The global prevalence of homocystinuria is estimated to be 0.82:100,000 based on clinical records and 1.09:100,000 by newborn screening.
• In the United States, homocystinuria affects about one out of every 100,000 people.
• Classic homocystinuria affects between 1 in 200,000 and 335,000 people worldwide.
• Some countries appear to have a higher prevalence of the disease, including Ireland (1 in 65,000), Germany (1 in 17,800), Norway (1 in 6,400), and Qatar (1 in 1,800).

Drugs:

CYSTADANE (betaine anhydrous for oral solution) is a methylating drug used to treat homocystinuria and reduce excessive homocysteine blood levels. Adult patients typically receive 6 grams per day orally, divided into two doses of 3 grams each.

Pegtibatinase is an investigational PEGylated recombinant enzyme replacement therapy that aims to treat the fundamental cause of classical homocystinuria (HCU). In preclinical trials, pegtibatinase was shown to lower total homocysteine levels and enhance clinical indicators.

Pegtarviliase is a novel recombinant human enzyme designed to lower plasma levels of total homocysteine by digesting the amino acid homocysteine and its dimer. Pegtarviliase is now being explored in Phase 1/2 clinical research for the treatment of people with classic homocystinuria.

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

Competitive Landscape:

This report also provides a detailed analysis of the current homocystinuria 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 homocystinuria 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 homocystinuria market across the seven major markets in 2023 and what will it look like in 2034?
• What is the growth rate of the homocystinuria 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 homocystinuria across the seven major markets?
• What is the number of prevalent cases (2018-2034) of homocystinuria by age across the seven major markets?
• What is the number of prevalent cases (2018-2034) of homocystinuria by gender across the seven major markets?
• How many patients are diagnosed (2018-2034) with homocystinuria across the seven major markets?
• What is the size of the homocystinuria 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 homocystinuria?
• What will be the growth rate of patients across the seven major markets?
   

Homocystinuria: 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 homocystinuria 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 homocystinuria market?
• What are the key regulatory events related to the homocystinuria market?
• What is the structure of clinical trial landscape by status related to the homocystinuria market?
• What is the structure of clinical trial landscape by phase related to the homocystinuria market?
• What is the structure of clinical trial landscape by route of administration related to the homocystinuria market?