Overview:

The term “porphyria” encompasses a collection of eight disorders that impact both the skin and nervous system. Most of these conditions are hereditary, passed down through family lines. Porphyrins (natural chemical) play a crucial role in the production of heme, a component of hemoglobin, the protein responsible for transporting oxygen to the body’s organs and tissues. To transform porphyrins into heme, the body relies on eight enzymes. When there is an insufficient amount of any of these enzymes, porphyrins build up, leading to elevated levels in the body. Such high levels of porphyrins can cause significant issues, particularly affecting the nervous system and skin as observed in Acute intermittent porphyria (AIP). It is caused by low levels of porphobilinogen deaminase. The main symptoms of AIP include insomnia, anorexia, changes in urine color, myalgia, and urinary retention. The condition can lead to liver tumors (hepatocellular carcinoma) and kidney failure. Gastrointestinal, urinary tract, psychological, and neurological issues are also common symptoms. Medications such as Panhematin, Normosang, Givlaari, chlorpromazine, and hemin can be used for its treatment. AIP is considered a rare disease. The exact prevalence is not well-known, as many cases may go undiagnosed due to its variable and nonspecific symptoms. It is estimated that AIP affects fewer than 5 in 100,000 individuals in the general population. The prevalence may vary among different populations and regions

The treatment goal for acute attacks of Acute Intermittent Porphyria (AIP) is to decrease heme synthesis and reduce porphyrin precursors. High-dose intravenous 5% glucose in 0.9% sodium chloride solution is recommended for mild attacks. Avoid intravenous glucose in water (D5W, D10W) to prevent aggravating hyponatremia. Patients experiencing severe attacks, particularly those with severe neurological symptoms, should be administered hematin at a dosage of 4 mg/kg per day for a duration of 4 days. Givosiran (Givlaari) has received approval from the US Food and Drug Administration (FDA) for use in adults with acute hepatic porphyrias characterized by attacks induced by the enzyme 5-aminolevulinic acid synthase 1 (ALAS1). Givosiran is a small-interfering RNA that functions through RNA interference, causing degradation of aminolevulinate synthase 1 (ALAS1) messenger RNA (mRNA) in hepatocytes. This mechanism helps reduce elevated levels of liver ALAS1 mRNA. The treatment involves monthly subcutaneous injections. Symptomatic treatment involves the utilization of beta-blockers to manage tachycardia and prevent arrhythmia. For hypertensive crisis, beta-blockers, clonidine, or other recommended antihypertensives can be employed. To control nausea and vomiting, olanzapine, lorazepam, or prochlorperazine can be utilized. Seizures are treated with Gebapentin; as most anti-seizure medicine can be contraindicated.

SCOPE:

• To Provide a Comprehensive Analysis of the Global Market for Acute Intermittent Porphyria Treatments.
• To Provide Informative data on Current Medications, Therapies, and Management Approaches for AIP.
• To Provide Profiling of Leading Pharmaceutical Companies and their Dominance in the AIP Treatment Market.
• To Investigate Promising Novel Approaches in AIP Treatment Research and Development.
• To provide a Comparative Study of AIP Treatment Options Revealing Efficacy, Safety, and Cost-Effectiveness.
• To Provide an Analysis of Growth Drivers and Noteworthy Trends Shaping the AIP Treatment Market.
• To Address Barriers of Treatment Accessibility and Affordability for AIP Patients.
• To Provide Informative Data about the Advancements in Pipeline and Emerging AIP Treatment and Proactive Attack Prevention.
• To Provide an Outlook on Projected Market Growth and Potential Investment Opportunities in AIP Treatment.

Market Landscape Assessment

To effectively assess the class competition over the short to medium-long term, it is essential to possess a comprehensive grasp of the development pipeline. This detailed understanding plays a crucial role in strategically positioning a product within the market.

Company Profiles

The stakeholders involved in the development of treatment solutions for Acute Intermittent Porphyria  have been compiled, encompassing pertinent details such as financials, product portfolio, and recent advancement

Patent Analysis

Through patent analysis, one can gain invaluable insights into the innovative developments and emerging technologies related to Acute Intermittent Porphyria. This process facilitates a deeper understanding of novel treatment approaches and their potential in the field.

Clinical Trials Analysis

Over the past five years, several drugs/therapies evaluated in clinical trials across various regions have progressed to advanced stages of development or have reached maturity.

Distribution by Key Geographical Areas

Distribution of Clinical Trials by Status

Distribution of Clinical Trials by Phase of Development

Market Forecast

The market is poised for substantial growth over the next decade due to the approval of multiple drug candidates and promising clinical outcomes. As a result, it is now distributed across various segments to accommodate the increasing demand.

Competitive Landscape

The presence of numerous companies operating in the field of Acute Intermittent Porphyria can lead to potential competitors vying to gain a dominant position in the market.

Report Highlights:

• This report provides a Comprehensive Analysis of Acute Intermittent Porphyria Treatment Market Dynamics and Growth Potential.
• This report helps in Capitalizing on Opportunities by Exploring Innovative Glucose and Heme-based Treatments’ Market Penetration for Porphyria Precursors.
• This report explores Future-Proofing Strategies of Assessing the Market Viability of Liver Transplantation in Severe AIP Cases.
• This report helps in Seizing the Early Mover Advantage by Emphasizing on the Impact of Timely Diagnosis on Market Penetration and Improved AIP Treatment Outcomes.
• This reports keeps a watchful Tracking on Current Studies and Projecting Market Potential of Promising Therapeutic Advancements in AIP Treatment.
• This report helps in Identifying the Market Demand and Growth Opportunities by conducting a thorough research on drug history and its efficacies and only then giving a forecast.
• This report will provide you an insight into projected market growth and potential investment opportunities in AIP treatment for 10 years

Report Insights:

• Acute Intermittent Porphyria Drug and Patent insights
• Acute Intermittent Porphyria Market Opportunity
• Size and trends for the Acute Intermittent Porphyria market
• Acute Intermittent Porphyria Pipeline and emerging treatment analysis
• Clinical trial analysis for the drugs used in Acute Intermittent Porphyria
• Company profiles of key players in the Acute Intermittent Porphyria

Research Methodology:

In order to give the most precise estimations and forecasts, Wissen Research uses an extensive and iterative research approach that is focused on reducing deviation. The company blends top-down and bottom-up methodologies for market segmentation and quantitative estimation. In addition, data triangulation, which examines the market from three separate angles, is a recurrent topic present in all of our research studies. Important components of the approach used for all of our studies include the following.:

Preliminary data mining

On a wide scale, unprocessed market data is collected. Continuous data filtering makes sure that only verified and authenticated sources are taken into account. Additionally, data is extracted from a wide range of reports in our repository and from a number of reputable premium databases. We gather information from raw material suppliers, distributors, and purchasers to help with this since understanding the entire value chain is crucial for a thorough understanding of the market.

Surveys, technical symposia, and trade magazines are used to gather information on technical concerns and trends. Technical information focusing on white space and freedom of movement is also obtained from an intellectual property standpoint. Additionally, information on the industry’s drivers, constraints, and pricing patterns is obtained. As a result, a variety of original data are included in the material that is then cross-validated and certified with published sources.

Statistical model

We use simulation models to generate our market projections and estimates. Every study receives a special model that is tailored to it. Data for market dynamics, the technology environment, application development, and pricing patterns are gathered and supplied into the model all at once for analysis. The relative relevance of these factors is investigated, and their impact on the forecast period is assessed, using correlation, regression, and time series analysis. The process of market forecasting combines technological analysis with economic strategies, practical business acumen, and subject expertise.

Econometric models are frequently used for short-term forecasting, but technology market models are typically employed for long-term forecasting. These are based on a confluence of the business environment, regulatory environment, economic projection, and technical landscape. In order to develop global estimates, it is preferable to estimate markets from the bottom up by integrating data from key regional markets. This is required to ensure accuracy and a complete comprehension of the subject. Among the variables taken into account for forecasting are:

• Market drivers and restraints, as well as the current and anticipated effects of each

   scenario for raw materials and supply versus pricing patterns

Regulations and anticipated developments

• Present capacity and anticipated capacity growth to 2033

We give these criteria weights and use weighted average analysis to assess their market influence in order to calculate the anticipated market growth rate

Data Collection Matrix:

1.     Executive Summary

1.1 Key Findings

1.2 Market Overview

1.3 Market Insights

2.     Introduction

2.1 Research Scope

2.2 Methodology

2.3 Report Objectives

2.4 Data Sources

 3. Disease Overview

3.1 Introduction to Acute Intermittent Porphyria (AIP)

3.2 Causes and Risk Factors

3.3 Symptoms and Clinical Presentation

3.4 Diagnosis and Diagnostic Techniques

4. Market Landscape

4.1 Market Size and Forecast by Type of AIP, 2023-2033 (USD Million)

4.1.1 Sporadic AIP (Type 1)

4.1.2 Familial AIP (Type 2)

4.2 Market Size and Forecast by Age Group, 2023-2033 (USD Million)

4.2.1 Adults

4.2.2 Pediatric Population

4.3 Market Size and Forecast by Region, 2023-2033 (USD Million)

4.3.1 North America

4.3.2 Europe

4.3.3 Asia Pacific

4.3.4 Latin America

4.3.5 Middle East & Africa

4.3.6 Market Trends and Outlook

5. Patent Analysis

5.1 Top Assignee

5.2 Geography focus of top Assignees

5.3 Medications and Drug Classes

5.4 Legal Status

5.5 Geography

5.6 Assignee Segmentation

5.7 Network analysis of the top collaborating entities in AIP treatments patent applications

5.8 Technology Evolution

5.9 Key Patents

5.10 Application trend

5.11 Application and Issued Trend

5.12 Key technology

6. Product Analysis

6.1 Current Treatment Options

6.1.1 Approved drugs

6.1.2 Pipeline drugs

6.2 Emerging Therapies and Research Developments (by product analysis and scientific analysis)

6.3 Medications and Drug Classes

6.4 Therapeutic activity of drugs

7. Clinical trial Analysis

7.1          Chapter Overview

7.2          Analysis by Trial Registration Year

7.3          Analysis by Phase of Development

7.4          Analysis by Number of Patients Enrolled

7.5          Analysis by Status of Trial

7.6          Analysis by Study Design

7.7          Analysis by Type of Product

7.8          Analysis by Geography

7.9          Analysis by Key Sponsors / Collaborators

8. Market Dynamics

8.1 Market Drivers

8.2 Market Restraints

8.3 Market Opportunities

8.4 Challenges and Future Prospects

9. Competitive Landscape

9.1 Market Share Analysis

9.2 Company Profiles

9.2.1 Recordati Rare Diseases

9.2.2 CLINUVEL Pharmaceuticals

9.2.3 Siemens

9.2.4 Alnylam Pharmaceuticals

9.2.5 Sysmex Corporation

9.3 Competitive Strategies and Developments

10. Regulatory and Reimbursement Landscape

10.1 Regulatory Approvals

10.2 Reimbursement Policies

11. Market Outlook and Future Trends

12. Conclusion

13. Appendix

13.1 List of Abbreviations

13.2 Research Methodology

13.3 Data Sources

13.4 Disclaimer

LIST OF FIGURES

LIST OF GRAPHS

LIST OF TABLES