Pyruvate Kinase Deficiency Market

Market Overview:

The 7 major pyruvate kinase deficiency markets are expected to exhibit a CAGR of 3.99% during 2024-2034.

The pyruvate kinase deficiency market has been comprehensively analyzed in IMARC's new report titled "Pyruvate Kinase Deficiency Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Pyruvate kinase deficiency refers to a genetic disorder that affects the red blood cells (erythrocytes) in the body. It is caused by changes (mutations) in the PKLR gene, resulting in a lack of the enzyme pyruvate kinase. People with this illness are often affected by chronic hemolytic anemia, a condition in which red blood cells prematurely undergo hemolysis, causing their shortage in the body. Indications of pyruvate kinase deficiency might vary significantly from person to person. Some of the common symptoms include an enlarged spleen, shortness of breath, yellowish skin, dark-colored urine, high concentrations of iron, weakness, etc. The diagnosis of this disease typically involves a full patient and family history, a thorough clinical evaluation, and a series of specialized tests. Furthermore, the medical practitioner may perform lab tests to determine the presence and cause of anemia. A low hemoglobin and haptoglobin level, along with an elevated unconjugated bilirubin level and reticulocyte count in the blood, are all indicative of confirmed disease in patients.

The rising prevalence of inherited genetic conditions on account of gene abnormalities, mutations in the DNA, and chromosomal irregularities is primarily driving the pyruvate kinase deficiency market. Furthermore, the widespread adoption of folic acid supplements to produce new red blood cells and reduce the risk of complications associated with the disorder is also augmenting the market growth. Apart from this, the inflating utilization of phototherapy in infants with pyruvate kinase deficiency, since it lowers the total serum bilirubin concentration without any likelihood of neurotoxicity, is further creating a positive outlook for the market. Additionally, several key players are investing in R&D activities for the launch of novel disease-modifying gene therapies, which allow the replacement of a mutated gene with a healthy one. This, in turn, is acting as another significant growth-inducing factor. Moreover, the increasing demand for an allosteric pyruvate kinase activator that works by upregulating the activity of erythrocyte pyruvate kinase and enhancing the lifespan of red blood cells in the body is expected to drive the pyruvate kinase deficiency market in the coming years.

IMARC Group's new report provides an exhaustive analysis of the pyruvate kinase deficiency market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report, the United States has the largest patient pool for pyruvate kinase deficiency and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the pyruvate kinase deficiency market in any manner.

Recent Developments:

• In May 2023, Rocket Pharmaceuticals reported that RP-L301, the company's experimental lentiviral-based gene therapy for pyruvate kinase deficiency, has been designated as a Regenerative Medicine Advanced Therapy (RMAT) by the United States Food and Drug Administration. RMAT designation was given based on strong safety and efficacy outcomes from the ongoing Phase 1 RP-L301 clinical trial, as well as the potential to cure a life-threatening illness for which no therapy is presently available.

Key Highlights:

• Pyruvate kinase deficiency is the most prevalent cause of chronic hereditary non-spherocytic hemolytic anemia, with a prevalence estimated to be between 1:20,000 and 1:300,000 in Caucasian populations.
• It occurs all around the world, but it is more frequent among people of northern European origin.
• Pyruvate kinase deficiency affects around one in every 20,000 people of European heritage.
• The condition is found in the majority of affected people during childhood, but in mild cases, it may not be discovered until late age.
• Pyruvate kinase deficiency is more common among Pennsylvania's Old Order Amish community.

Drugs:

PYRUKYND is a prescription medication used to treat low red blood cell counts resulting from early red blood cell breakdown (hemolytic anemia) in adults suffering from pyruvate kinase deficiency. It is a pyruvate kinase activator that binds to the pyruvate kinase tetramer and increases pyruvate kinase activity.

RPL-301 is a gene-modified cell therapy being developed for the treatment of pyruvate kinase deficiency. The treatment candidate is patient-derived immature bone marrow cells (CD34+) that have been genetically engineered using a lentivirus to express the pyruvate kinase (PKLR) enzyme. It is delivered via the intravenous method.

Time Period of the Study

• Base Year: 2024
• Historical Period: 2019-2024
• Market Forecast: 2024-2034
   

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan
   

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the pyruvate kinase deficiency market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the pyruvate kinase deficiency market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current pyruvate kinase deficiency marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the pyruvate kinase deficiency market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2024 and how are they expected to perform till 2034?
• What was the country-wise size of the pyruvate kinase deficiency market across the seven major markets in 2024 and what will it look like in 2034?
• What is the growth rate of the pyruvate kinase deficiency market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?
   

Epidemiology Insights

• What is the number of prevalent cases (2018-2034) of pyruvate kinase deficiency across the seven major markets?
• What is the number of prevalent cases (2018-2034) of pyruvate kinase deficiency by age across the seven major markets?
• What is the number of prevalent cases (2018-2034) of pyruvate kinase deficiency by gender across the seven major markets?
• How many patients are diagnosed (2018-2034) with pyruvate kinase deficiency across the seven major markets?
• What is the size of the pyruvate kinase deficiency patient pool (2019-2024) across the seven major markets?
• What would be the forecasted patient pool (2024-2034) across the seven major markets?
• What are the key factors driving the epidemiological trend of pyruvate kinase deficiency?
• What will be the growth rate of patients across the seven major markets?
   

Pyruvate Kinase Deficiency: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for pyruvate kinase deficiency drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the pyruvate kinase deficiency market?
• What are the key regulatory events related to the pyruvate kinase deficiency market?
• What is the structure of clinical trial landscape by status related to the pyruvate kinase deficiency market?
• What is the structure of clinical trial landscape by phase related to the pyruvate kinase deficiency market?
• What is the structure of clinical trial landscape by route of administration related to the pyruvate kinase deficiency market?