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Homocystinuria Therapeutics Market: Introduction

  • Homocystinuria is a disorder of sulfur metabolism pathway caused by deficiency of cystathionine β-synthase (CBS). Increased homocysteine results in various vascular and neurological complications. Thromboembolism, affecting both large and small arteries and veins, is the major cause of morbidity and mortality. Intellectual deficiency rarely manifests before the first to second year of life. Clinically significant psychiatric illness is found in 51% of cases.

Key Drivers and Restraints of Global Homocystinuria Therapeutics Market

  • High demand for disease specific novel treatment can act as a major driver of the global market. Population studies suggest that the global prevalence of homocystinuria in the general population is 1 in 344,000. Prevalence may be higher in different countries. Europe has reported higher cases than North America and Asia Pacific. In Ireland, the prevalence has been estimated to be 1 in 65,000. In Germany, the prevalence has been estimated at 1 in 17,500.
  • However, limited patient pool for clinical trials and product marketing is likely to hamper the growth of the global market in the near future. Clinical trials for homocystinuria therapeutics are often smaller than those of other drugs and require the development of efficient trial designs relevant to small populations to gain the most information from the available data. Further progress is needed in trial design aiming to assess medicines in a cost-effective, rigorously controlled manner. These factors are likely to hamper the growth of the global market.

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Pyridoxine to Dominate Global Homocystinuria Therapeutics Market

  • In terms of drug treatment, the global homocystinuria therapeutics market can be classified into pyridoxine, betaine, and others
  • Pyridoxine is the first-line therapeutic prescribed for homocystinuria treatment. In pyridoxine non-responsive individuals, the recommended treatment is a methionine-restricted, cystine-supplemented diet in combination with the pyridoxine, folic acid, and vitamin B12 supplementation. In May 2018, Duchesnay USA launched Bonjesta (doxylamine succinate and pyridoxine hydrochloride), multilayer extended-release tablets

Online Pharmacies Segment Witness Highest Growth

  • Based on distribution channel, the global homocystinuria therapeutics market can be divided into hospital pharmacies, retail pharmacies, and online pharmacies
  • The retail pharmacies segment accounted for the largest share of the global homocystinuria therapeutics market in 2019. However, the online pharmacies segment is expected to expand at the highest CAGR from 2020 to 2030.
  • Retail pharmacies, presently, use cloud-based inventory management to share information across several locations, automatically re-order stock, and review past transactions. In Asia, the majority of drug retailing in carried out via hospital and retail pharmacy channels. On average, retail pharmacies account for 71% of sales of therapeutic drugs, while hospital pharmacies account for 29% across the region.

North America to Lead Global Cystinuria Treatment Market

  • In terms of region, the global homocystinuria therapeutics market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa
  • In terms of revenue, Europe dominated the homocystinuria therapeutics market in 2019, followed by North America. However, the market in Asia Pacific is projected to expand at the highest CAGR during the forecast period.
  • Rise in cases of homocystinuria, surge in usage, and increase in awareness about therapeutics available are the key factors boosting the growth of the homocystinuria therapeutics market in Europe. The disorder is more common in Ireland, Germany, and Norway. Several studies in four countries in Europe suggested that the prevalence of homocystinuria due to CBS deficiency is as high as 1 in 6,400 to 1 in 20,000.

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Key Players Operating in Global Homocystinuria Therapeutics Market

The global homocystinuria therapeutics market was highly fragmented in 2019. Key players operating in the global market are:

  • HuaZhong Pharmaceutical
  • DSM N.V.
  • Hospira Healthcare Ulc
  • Mylan Pharmaceuticals
  • Alveda Pharmaceuticals, Inc.
  • Nutreco N.V.
  • Kao Corporation
  • Amino GmbH

Global Homocystinuria Therapeutics Market: Research Scope

Global Homocystinuria Therapeutics Market, by Drug Treatment

  • Pyridoxine
  • Betaine
  • Others (folate, cobalamin, etc.)

Global Homocystinuria Therapeutics Market, by Distribution Channel

  • Hospital Pharmacies
  • Retail Pharmacies
  • Online Pharmacies

Global Homocystinuria Therapeutics Market, by Region

  • North America
    • U.S.
    • Canada
  • Europe
    • Germany
    • U.K.
    • France
    • Italy
    • Spain
    • Rest of Europe
  • Asia Pacific
    • China
    • India
    • Japan
    • Australia & New Zealand
    • Rest of Asia Pacific
  • Latin America
    • Brazil
    • Mexico
    • Rest of Latin America
  • Middle East & Africa
    • GCC Countries
    • South Africa
    • Rest of Middle East & Africa

This study by TMR is all-encompassing framework of the dynamics of the market. It mainly comprises critical assessment of consumers' or customers' journeys, current and emerging avenues, and strategic framework to enable CXOs take effective decisions.

Our key underpinning is the 4-Quadrant Framework EIRS that offers detailed visualization of four elements:

  • Customer Experience Maps
  • Insights and Tools based on data-driven research
  • Actionable Results to meet all the business priorities
  • Strategic Frameworks to boost the growth journey

The study strives to evaluate the current and future growth prospects, untapped avenues, factors shaping their revenue potential, and demand and consumption patterns in the global market by breaking it into region-wise assessment.

The following regional segments are covered comprehensively:

  • North America
  • Asia Pacific
  • Europe
  • Latin America
  • The Middle East and Africa

The EIRS quadrant framework in the report sums up our wide spectrum of data-driven research and advisory for CXOs to help them make better decisions for their businesses and stay as leaders.

Below is a snapshot of these quadrants.

1. Customer Experience Map

The study offers an in-depth assessment of various customers’ journeys pertinent to the market and its segments. It offers various customer impressions about the products and service use. The analysis takes a closer look at their pain points and fears across various customer touchpoints. The consultation and business intelligence solutions will help interested stakeholders, including CXOs, define customer experience maps tailored to their needs. This will help them aim at boosting customer engagement with their brands.

2. Insights and Tools

The various insights in the study are based on elaborate cycles of primary and secondary research the analysts engage with during the course of research. The analysts and expert advisors at TMR adopt industry-wide, quantitative customer insights tools and market projection methodologies to arrive at results, which makes them reliable. The study not just offers estimations and projections, but also an uncluttered evaluation of these figures on the market dynamics. These insights merge data-driven research framework with qualitative consultations for business owners, CXOs, policy makers, and investors. The insights will also help their customers overcome their fears.

3. Actionable Results

The findings presented in this study by TMR are an indispensable guide for meeting all business priorities, including mission-critical ones. The results when implemented have shown tangible benefits to business stakeholders and industry entities to boost their performance. The results are tailored to fit the individual strategic framework. The study also illustrates some of the recent case studies on solving various problems by companies they faced in their consolidation journey.

4. Strategic Frameworks

The study equips businesses and anyone interested in the market to frame broad strategic frameworks. This has become more important than ever, given the current uncertainty due to COVID-19. The study deliberates on consultations to overcome various such past disruptions and foresees new ones to boost the preparedness. The frameworks help businesses plan their strategic alignments for recovery from such disruptive trends. Further, analysts at TMR helps you break down the complex scenario and bring resiliency in uncertain times.

The report sheds light on various aspects and answers pertinent questions on the market. Some of the important ones are:

1. What can be the best investment choices for venturing into new product and service lines?

2. What value propositions should businesses aim at while making new research and development funding?

3. Which regulations will be most helpful for stakeholders to boost their supply chain network?

4. Which regions might see the demand maturing in certain segments in near future?

5. What are the some of the best cost optimization strategies with vendors that some well-entrenched players have gained success with?

6. Which are the key perspectives that the C-suite are leveraging to move businesses to new growth trajectory?

7. Which government regulations might challenge the status of key regional markets?

8. How will the emerging political and economic scenario affect opportunities in key growth areas?

9. What are some of the value-grab opportunities in various segments?

10. What will be the barrier to entry for new players in the market?

Note: Although care has been taken to maintain the highest levels of accuracy in TMR’s reports, recent market/vendor-specific changes may take time to reflect in the analysis.

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Homocystinuria Therapeutics Market