The novel treatment approaches, which range from a small molecule pyruvate kinase activator to gene therapy, may transform the way in which Pyruvate Kinase Deficiency is managed in the future.
As per DelveInsight, the Pyruvate Kinase Deficiency market is expected to grow in the coming years owing to the rise in the number of prevalent cases of Pyruvate Kinase Deficiency patients in 7MM, improvement in the diagnosis methodologies, incremental healthcare spending across the world, and growth in research and development (R&D) activities into new treatments.
DelveInsight’s “Pyruvate Kinase Deficiency (PKD) Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Pyruvate Kinase Deficiency Market size, share, and trends in the seven major market (7MM) (i.e., the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan).
The Pyruvate Kinase Deficiency market report covers emerging drugs, current treatment practices, market share of the individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Pyruvate Kinase Deficiency (PKD): An Overview
PKD is caused by alterations (mutations) in the PKLR gene, which lead to a deficiency of the enzyme pyruvate kinase. These genetic alterations are inherited in an autosomal recessive manner. Pyruvate kinase is an enzyme that helps cells turn to sugar (glucose) into energy (called adenosine triphosphate, ATP) in a process called glycolysis. Red cells rely on this process for energy, and so, pyruvate kinase deficiency leads to a deficiency in energy and to premature red cell destruction (haemolysis). Instead of lasting 120 days, red cells with pyruvate kinase deficiency last only a few days to weeks.
Mild cases require no treatment. People with severe anaemia may need blood transfusions. In newborns with dangerous levels of jaundice, a health care provider may recommend an exchange transfusion.
Pyruvate Kinase Deficiency (PKD) Market Key Facts
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Pyruvate Kinase Deficiency is a rare disorder that affects both men and women. The frequency of the disorder is unknown, although one estimate suggests that approximately 1 in 20,000 Caucasian people develop the disorder.
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Pyruvate Kinase Deficiency has a worldwide distribution, although it is more prevalent in Northern Europe, Japan and China. The global incidence is around five cases per population of one million. In the United States, the incidence is 1 in 20 000, and most cases are identified via prenatal genetic screening. The estimated prevalence of the disease is 51 per million population.
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As per the NORD, in clinical practice, the frequency is closer to 1 in 1,000,000 people. PKD has been identified most commonly in Europe. However, rare disorders like PKD often go misdiagnosed or undiagnosed making it difficult to determine their true frequency in the general population.
Pyruvate Kinase Deficiency (PKD) Market
The Pyruvate Kinase Deficiency Market to exhibits growth owing to the increasing awareness about available treatments in the market, a better understanding of disease pathogenesis, and the development of novel therapeutics, among others.
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted Pyruvate Kinase Deficiency market size by analyzing the impact of current and emerging pipeline therapies. It also provides a thorough assessment of the market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete detail of the Pyruvate Kinase Deficiency market trend for each marketed drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MoA), Route of Administration (RoA), molecule types, competition with other therapies, brand value, and their impact on the market.
Pyruvate Kinase Deficiency (PKD) Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pool, future trends, and and views of key opinion leaders.
Pyruvate Kinase Deficiency (PKD) Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the rate of uptake of the potential drugs recently launched in the Pyruvate Kinase Deficiency market or expected to be launched during the study period. The analysis covers Pyruvate Kinase Deficiency market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Pyruvate Kinase Deficiency Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyses recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
Key Developments in the Pyruvate Kinase Deficiency (PKD) Therapeutics Market
To redress the current issues, very few companies are working toward this indication to meet the market’s needs. Pipeline drugs in clinical development are designed to address the major unmet needs in Pyruvate Kinase Deficiency treatment.
• On Sept. 16, 2022, Agios Pharmaceuticals (AGIO) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) had adopted a positive opinion and recommended the granting of a marketing authorization for its lead therapy PYRUKYND (mitapivat) for the treatment of pyruvate kinase (PK) deficiency in adult patients.
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Pyruvate Kinase Deficiency (PKD) Therapeutics Analysis
Some of the Common Supportive Treatments for Pyruvate Kinase Deficiency include Simple transfusion, Supplemental folic acid and vitamins, Chelation therapy, Phototherapy, and Splenectomy. The current pipeline for Pyruvate Kinase Deficiency (PKD) does not hold many significant products, it has very few products that certain key players are developing.
Some of the key companies in the Pyruvate Kinase Deficiency Market include:
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Agios Pharmaceuticals
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Rocket Pharmaceuticals
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Centogene AG
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Rostock
And many others.
Pyruvate Kinase Deficiency Therapies covered in the report include:
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Mitapivat
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RP-L301
And many more.
Further Emerging Drugs & Key Companies are Covered in the Report. Download the Sample PDF to Learn More –
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Pyruvate Kinase Deficiency Competitive Intelligence Analysis
4. Pyruvate Kinase Deficiency Market Overview at a Glance
5. Pyruvate Kinase Deficiency Disease Background and Overview
6. Pyruvate Kinase Deficiency Patient Journey
7. Pyruvate Kinase Deficiency Epidemiology and Patient Population (In US, EU5, and Japan)
8. Pyruvate Kinase Deficiency Treatment Algorithm, Current Treatment, and Medical Practices
9. Pyruvate Kinase Deficiency Unmet Needs
10. Key Endpoints of Pyruvate Kinase Deficiency Treatment
11. Pyruvate Kinase Deficiency Marketed Products
12. Pyruvate Kinase Deficiency Emerging Drugs and Latest Therapeutic Advances
13. Pyruvate Kinase Deficiency Seven Major Market Analysis
14. Attribute Analysis
15. Pyruvate Kinase Deficiency Market Outlook (In US, EU5, and Japan)
16. Pyruvate Kinase Deficiency Access and Reimbursement Overview
17. KOL Views on the Pyruvate Kinase Deficiency Market
18. Pyruvate Kinase Deficiency Market Drivers
19. Pyruvate Kinase Deficiency Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance.
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