Pyruvate Kinase Deficiency (PKD) Market to Evolve with Dynamic Clinical and Commercial Development Activities in Therapeutics Segment | Rocket Pharmaceutical (RP-L301), Agios Pharmaceuticals (PYRUKYND)

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 and Share in the 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, treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current treatment practice/algorithm, market drivers & barriers, and unmet medical needs to curate the best of opportunities and assess the underlying potential of the market.

Pyruvate Kinase Deficiency (PKD): An Overview
Pyruvate kinase deficiency 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).

Pyruvate Kinase Deficiency Market Key Facts
According to NORD, Pyruvate Kinase Deficiency 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.
As per the assessment, Pyruvate Kinase Deficiency affects males and females equally.
As per DelveInsight, an increase in the prevalence of pyruvate kinase deficiency, along with the expected launch of emerging therapies, will boost the Pyruvate Kinase Deficiency Market in the forecast period [2022-2032].
Currently, RP-L301 is the most anticipated emerging therapy that is awaiting launch. This emerging therapy is in the mid-phases of clinical trials.

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 therapies in the market. It also provides a detailed assessment of the market drivers & barriers, unmet needs, and emerging technologies.

The report gives complete detail of the Pyruvate Kinase Deficiency market trend for each marketed drug and late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action (MoA), competition with other therapies, brand value, their impact on the market and view of the key opinion leaders.

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Pyruvate Kinase Deficiency (PKD) Epidemiology
The epidemiology section covers detailed insights into the historical and current Pyruvate Kinase Deficiency patient pool and forecasted trends for every seven major countries (7MM) from 2019 to 2032.

Pyruvate Kinase Deficiency (PKD) Epidemiology Segmentation
Total Prevalent Cases of Pyruvate Kinase Deficiency
Diagnosed Prevalent Cases of Pyruvate Kinase Deficiency
Age-specific Diagnosed Prevalent cases of Pyruvate Kinase Deficiency

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Pyruvate Kinase Deficiency (PKD) Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Pyruvate Kinase Deficiency market or expected to get launched in the market during the study period. The analysis covers Pyruvate Kinase Deficiency market uptake by drugs, patient uptake by therapies, and sales of each drug.

Report's Drugs Uptake section helps in understanding the drugs with the most rapid uptake and reasons behind the maximal use of new drugs and allows the comparison of the drugs based on market share and size, which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

The report also covers the Pyruvate Kinase Deficiency Pipeline Development Activities and provides valuable insights about different therapeutic candidates in Phase II and Phase III stages and the key companies in the market involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

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Pyruvate Kinase Deficiency (PKD) Therapeutics Assessment
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, such as Rocket Pharmaceutical, Agios Pharmaceuticals, and others, are developing a drug for the treatment of Pyruvate Kinase Deficiency.

Rocket Pharmaceutical is working on RP-L301, a gene therapy product containing autologous genetically modified CD34+ hematopoietic stem cells containing the corrected PKD gene. In May 2022, Rocket Pharmaceuticals announced positive clinical updates from its Phase II pivotal trial for Fanconi Anemia (FA) and Phase I trials for Danon Disease and Pyruvate Kinase Deficiency (PKD) at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT).

On November 11, 2022, Agios Pharmaceuticals, Inc. (Nasdaq: AGIO) announced that the European Commission (EC) has granted marketing authorization for PYRUKYND® for the treatment of PK deficiency in adult patients.

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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
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 Therapies
13. Pyruvate Kinase Deficiency Seven Major Market Analysis
14. Attribute Analysis
15. Pyruvate Kinase Deficiency Market Outlook (7 major markets)
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 is not exhaustive; the final content may vary.

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Email: info@delveinsight.com
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