Hypophosphatasia Treatment: Introduction
- Hypophosphatasia (HPP) is a rare genetic disorder characterized by the abnormal development of bones and teeth. These abnormalities occur due to defective mineralization such as calcium and phosphorus. These minerals are required for proper hardness and strength. Defective mineralization results in bones becoming soft and more prone to fracture.
- Hypophosphatasia symptoms can vary greatly from one person to another, sometimes even among members of the same family. HPP is caused by mutations in the tissue nonspecific alkaline phosphatase (ALPL) gene, also known as TNSALP gene. Such mutations lead to low activity of the tissue nonspecific alkaline phosphatase enzyme. Depending on the specific form, HPP can be inherited in an autosomal recessive or autosomal dominant manner.
Key Drivers of Global Hypophosphatasia Treatment Market
- Increasing number of patients with hypophosphatasia, family history of hypophosphatasia, strong product pipeline, and increasing financial support to researchers for developing new innovative drugs for treatment of hypophosphatasia are likely to drive the hypophosphatasia market during the forecast period. In addition, increase in special designation from the regulatory authorities to offer a treatment to the patients as quickly as possible is enhancing the market.
- According to the National organization for Rare Disorders, the overall incidence and prevalence of hypophosphatasia is unknown, but in Canada, the severe form of hypophosphatasia affects approximately 1 in 100,000 live births.
- In other hand, patent expiration of major brands and less treatment options available are likely to restrain the growth of this market
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Odontohypophosphatasia Segment Expected to be Most Promising Segment
- Based on type, the global hypophosphatasia treatment market can be segmented into odontohypophosphatasia, pseudohypophosphatasia, and others
- The odontohypophosphatasia segment accounted for the highest share of the hypophosphatasia treatment market in 2019. This segment is expected to dominate the hypophosphatasia treatment market due to high number of patients with odontohypophosphatasia, which is characterized by loss of premature teeth in childhood or loss of teeth in adulthood.
Medication Segment to Witness Significant Growth
- In terms of treatment type, the global hypophosphatasia treatment market can be divided into medication and surgery. The medication segment is further divided into asfotase alfa, non-steroidal anti-inflammatory drugs, and others.
- The medication segment generated the highest revenue in 2019 and is anticipated to witness growth during the forecast period. This can be attributed to launch of products for treatment of hypophosphatasia from major manufacturers. In 2015, the U.S. Food and Drug Administration (FDA) approved Strensiq (asfotase alfa) as the first approved medical treatment for infantile, perinatal, and juvenile-onset HPP.
Hospitals Pharmacies Segment to be Most Promising Segment
- In terms of distribution channel, the global hypophosphatasia treatment market can be divided into hospital pharmacies, retail pharmacies, and online sales. The hospital pharmacies segment generated the highest revenue in 2019 and is anticipated to witness growth during the forecast period due to most of major products are prescription products, which is available in hospital pharmacies.
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North America to Dominate Global Hypophosphatasia Treatment Market
- In terms of region, the global hypophosphatasia treatment market can be categorized into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa
- North America dominated the global hypophosphatasia treatment market in 2019 and is expected to maintain its leading position during the forecast period. This dominance of the region can be attributed to increasing research & development activity from major market players, increase in product offerings, greater affordability, and rise in demand for early treatment.
- The market in Asia Pacific is projected to register highest growth during the forecast period, owing to increase in adoption of new products and rise in demand for hypophosphatasia treatment in emerging countries such as India and China
Key Manufacturers Operating in Global Market
The global hypophosphatasia treatment market was highly fragmented in 2019. Key manufacturers operating in the global market are:
- Kirin Holdings Company
- Vericel Corporation
- Mereo BioPharma Group plc
- Novartis AG
- AM?Pharma B.V.
- Bayer AG
- Pfizer, Inc.
Hypophosphatasia Treatment Market: Research Scope
Hypophosphatasia Treatment Market, by Type
Hypophosphatasia Treatment Market, by Treatment Type
- Asfotase Alfa
- Non-steroidal Anti-inflammatory Drugs
Hypophosphatasia Treatment Market, by Distribution Channel
- Hospital Pharmacies
- Retail Pharmacies
- Online Sales
Hypophosphatasia Treatment Market, by Region
- North America
- Rest of Europe
- Asia Pacific
- Australia & New Zealand
- Rest of Asia Pacific
- Latin America
- 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
- 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.