Companies across the globe are diligently working toward the development of novel treatment therapies, with a considerable amount of success over the years. Key players like Rocket Pharmaceutical, Agios Pharmaceuticals, and others are developing drugs to treat pyruvate kinase deficiency. RP-L301 is the most anticipated emerging therapy in the PKD therapeutics landscape.
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, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The Pyruvate Kinase Deficiency market report covers emerging drugs, current treatment practices, market share of 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
Pyruvate kinase deficiency (PKD) is a rare genetic blood disorder characterized by low levels of an enzyme called pyruvate kinase, used by red blood cells. PKD is caused by alterations (mutations) in the PKLR gene, which lead to a deficiency of the enzyme pyruvate kinase. Without pyruvate kinase, red blood cells break down too easily, resulting in low levels of these cells (hemolytic anemia). PKD is passed down as an autosomal recessive trait; this means a child must receive a non-working gene from each parent to develop the disorder. Many different types of enzyme-related defects of the red blood cell can cause hemolytic anemia, and PKD is the second most common cause after glucose-6-phosphate dehydrogenase (G6PD) deficiency.
Pyruvate kinase deficiency (PKD) is found in people of all ethnic backgrounds. But, certain populations, such as the Amish, are more likely to develop the condition. 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 Key Facts
According to NORD, PKD is a rare disorder that affects both men and women. The frequency of the condition is unknown, although one estimate suggests that approximately 1 in 20,000 Caucasian people develop the condition. In clinical practice, the frequency is closer to 1 in 1,000,000 people.
According to Grace et al. (2019), PKD exhibits autosomal recessive inheritance in nearly all cases and is widely distributed geographically. The frequency is not precisely defined, with a wide estimated prevalence of 3:1,000 000 to 1:20,000. Based on the qualitative and quantitative biochemical analysis, reported heterozygote frequencies among different populations range from 0.15% to 6%.
As per the study by Christensen et al. (2020) was deemed to be of high quality and useful for estimating PKD prevalence. The authors screened 153,830 live births in the US for high bilirubin; those with neonatal jaundice were subsequently tested for PKD. Five neonates were found to have PKD (prevalence of approximately 1 case per 30,000 births).
Pyruvate Kinase Deficiency (PKD) Market
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 thoroughly assesses 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 Assessment
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 views of key opinion leaders.
The Report Covers the Pyruvate Kinase Deficiency Epidemiology, Segmented as –
Total Prevalent Cases of Pyruvate Kinase Deficiency in the 7MM [2019–2032]
Diagnosed Prevalent Cases of Pyruvate Kinase Deficiency in the 7MM [2019–2032]
Age-specific Diagnosed Prevalent cases of Pyruvate Kinase Deficiency in the 7MM [2019–2032]
Pyruvate Kinase Deficiency (PKD) Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Pyruvate Kinase Deficiency market or expected to be launched during the study period. The analysis covers the 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.
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The Leading Companies in the Pyruvate Kinase Deficiency (PKD) Therapeutics Market Include:
And Many Others
Pyruvate Kinase Deficiency (PKD) Therapies Covered in the Report Include:
And Many More
The Report Covers the In-depth Assessment of the Emerging Drugs & Key Companies. Download the Sample Report to Learn More @
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 the 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
21. DelveInsight Capabilities
*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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