Alkaptonuria: Rare Form of Disease Shows Major Symptoms in Adulthood

  • Alkaptonuria is a rare form of a genetic metabolic disorder characterized by the accumulation of homogentisic acid in the body. This disease is also called black urine disease or black bone disease.
  • Affected people may have dark urine or urine that turns black when exposed to air. However, this change may not occur for several hours after urination and often goes unnoticed.
  • Black urine is main symptom in this disease which is present from birth. However, more symptoms can be observed in adulthood. The disorder is caused by mutation of the homogentisate 1, 2-dioxygenase (HGD) gene.
  • Symptoms of this disease include ochronosis, eye sclera discoloration, tendonitis, and long-standing conditions leading to arthritis
  • No medications are known to be useful in managing alkaptonuria. However, symptomatic relief can be provided by different classes of drugs. Also, several drugs are in investigational phase for the treatment of alkaptonuria.

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Key Drivers and Restraints of Global Alkaptonuria Drugs Market

  • Increase in new reported cases of alkaptonuria is the major factor responsible for the growth of the global alkaptonuria drugs market
  • For instance, according to an article published in the Journal of Rare Diseases on December 2013, the worldwide prevalence of alkaptonuria is 1 case in 250,000–1,000,000 births and total 950 alkaptonuria patients was identified in 40 countries by the end of  2013
  • Rise in awareness about alkaptonuria disease and clinical investigational therapies for the disease are some of the major factors fuelling the growth of the global alkaptonuria market
  • Patient assistance programs and different support programs in terms of treatment and research run by different organizations for alkaptonuria have further contributed to the growth of the global alkaptonuria drugs market

Ineffective Treatment Methods Hamper Market Growth

  • Not much medications are available which can be fully described as effective options for the treatment for alkaptonuria. This unavailability of effective treatment options is hampering the market growth.
  • Inadequate knowledge of the disease in some developing countries can also restrain the market growth

Enzyme Inhibitors - Highly Promising Drug Segment

  • Based on drug class, the global alkaptonuria drugs market can be divided into vitamins, enzyme Inhibitors, and others
  • Enzyme inhibitors include mainly Nitisinone which is a highly promising drug candidate for the treatment of alkaptonuria. This segment is anticipated to be a highly lucrative segment in the next few years due to ongoing clinical trials and availability of branded and generic options in this category.
  • Vitamin C (ascorbic acid) is the major vitamin used for various metabolic processes. Mild antioxidant nature of vitamin C helps to retard the process of conversion of homogentisate to the polymeric material that is deposited in cartilaginous tissues.

Oral Route of Administration Dominated Global Market

  • In terms of route of administration, the global alkaptonuria drugs market can be bifurcated into oral and injectable
  • Oral route of administration dominated the global alkaptonuria drugs market due to accurate unit dosage forms by tablets & capsules in adults and higher preference in children for suspension

Online Pharmacies to be Promising Segment

  • On the basis of distribution channel, the global alkaptonuria drugs market is classified into hospital pharmacies, retail pharmacies, and online pharmacies
  • Easy availability of branded as well as generic drugs and easy access to retail pharmacies are some of the major factors responsible for the dominance of the retail pharmacies segment
  • The online pharmacies segment is expected to be a highly lucrative segment during the forecast period due to rise in preference of online purchase by people and better discounted rates available at these stores.

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North America to Lead Global Alkaptonuria Drugs Market

  • In terms of region, the global alkaptonuria drugs market can be divided into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa
  • Europe dominated the global alkaptonuria drugs market in 2018 due to higher number of reported cases of alkaptonuria in the region, ongoing clinical trials, and better awareness regarding disease & its management
  • For instance, according to an article published in the Journal of Rare Diseases on December 2013, total 206 patients in Slovakia and 90 patients in Germany were identified for alkaptonuria by the end of 2013
  • Asia Pacific is anticipated to be a lucrative market for alkaptonuria drugs during the forecast period due to higher alkaptonuria patient population in some countries such as India, growing awareness of disease, and rise in focus and presence of companies in the region due to major market opportunities in this region

Key Manufacturers Operating in Global Market

 Key manufacturers operating in the global market are:

  • CYCLE Pharmaceuticals Limited
  • Sobi, Inc.
  • Emerge Health
  • Menarini Australia Pty Ltd
  • uniQure N.V.
  • GlaxoSmithKline plc
  • Sanofi
  • Pfizer Inc.
  • Teva Pharmaceutical Industries Ltd.
  • Mylan N.V.

Global Alkaptonuria Drugs Market: Research Scope

Global Alkaptonuria Drugs Market, by Drug Class

  • Vitamins
  • Enzyme Inhibitors
  • Others

Global Alkaptonuria Drugs Market, by Route of Administration

  • Oral
  • Injectable
  • Others

Global Alkaptonuria Drugs Market, by Distribution Channel

  • Hospital Pharmacies
  • Retail Pharmacies
  • Online Pharmacies

Global Alkaptonuria Drugs 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.

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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.

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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?

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