DelveInsight’s “Fanconi Anemia Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Fanconi Anemia, historical and forecasted epidemiology as well as the Fanconi Anemia market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.

 

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Key Takeaways from the Fanconi Anemia Market Report

• In November 2024:- Foresee Pharmaceuticals Co. Ltd- This is a multi-center, Phase 1/2 study to determine the Optimal Biologic Dose (OBD) and to evaluate the safety, tolerability, PK, and preliminary activity of FP 045 when administered orally in young adult/adolescent and pediatric patients with Fanconi anemia. The study will enroll a total of 4 young adult/adolescent patients and a minimum of 8 and up to 12 pediatric patients with mild-moderate bone marrow failure who have not undergone hematopoietic cell transplant.
• In 2023, the combined diagnosed prevalent cases of Fanconi Anemia in the EU4 countries and the UK amounted to approximately 3 thousand. Over the study duration, a significant rise in cases is expected across all participating nations. Among the EU4 countries, Germany recorded the highest proportion of Fanconi Anemia cases, whereas Spain reported the fewest cases.
• The estimates show the Total Diagnosed Prevalent Cases of Fanconi Anemia in Japan was found to be around 1.1 thousand in 2023, which is estimated to increase by 2034.
• Based on DelveInsight’s analysis, experts categorized the Mutation-specific Cases of Fanconi Anemia according to specific mutations, including FANCA, FANCC+FANCG, FANCE+FANCF, and others. In 2023, the FANCA category accounted for the highest number of cases, approximately 2 thousand, in the United States.
• In 2023, cases of Comorbidity associated with Fanconi Anemia were categorized into Hematological disease, Hematological malignancy, and Solid tumors. The highest number of cases, totaling 2.8 thousand, were attributed to Hematological disease in the US.
• The leading Fanconi Anemia Companies such as Rocket Pharma, Foresee Pharmaceuticals, Jasper Therapeutics, Novartis, and others
• Promising Fanconi Anemia Therapies such as RP-L102, FP-045, PS191, Deferasirox, and others

 

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Fanconi Anemia Epidemiology Segmentation in the 7MM

• Total Fanconi Anemia Prevalence
• Prevalent Cases of Fanconi Anemia by severity
• Fanconi Anemia Gender-specific Prevalence
• Fanconi Anemia Diagnosed Cases of Episodic and Chronic

 

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Fanconi Anemia Emerging Drugs

• RP-L102 (lentiviral gene therapy): Rocket Pharmaceuticals

RP-L102 is a gene therapy under development for treating Fanconi Anemia, utilizing a lentiviral vector (LVV). This therapy carries the FANC-A gene within the PGK-FANCA-WPRE expression cassette, incorporating a phosphoglycerate kinase (PKG) promoter and an optimized woodchuck hepatitis virus post-transcriptional regulatory element (WPRE). Through an ex vivo process, hematopoietic stem cells are extracted and enriched using CD34+ selection. These genetically modified CD34+ enriched hematopoietic cells, either fresh or cryopreserved, are then reintroduced into patients to restore function. RP-L102 has received various designations, including ODD, RPDD, FTD, and RMAT designation in the United States, as well as ODD and PRIME status in Europe. Moreover, the EMA has classified this therapy as an ATMP.

 

• FP-045: Foresee Pharmaceuticals

FP-045 is a potent and specifically targeted activator of aldehyde dehydrogenase 2 (ALDH2), characterized by high solubility and oral availability. ALDH2 serves as a crucial mitochondrial regulator involved in metabolizing harmful aldehydes, particularly those generated during ethanol metabolism and oxidative stress. Moreover, FP-045 has demonstrated the ability to enhance ALDH2 activity in vitro within FANCA-deficient cells, offering protection against damage caused by exposure to reactive aldehydes.

 

Fanconi Anemia Market Outlook

Fanconi Anemia treatment encompasses various classifications within the disease spectrum. The primary focus of managing Fanconi Anemia involves the use of Androgen Therapy and Maintenance Therapies as required, with Androgen Therapy serving as the principal revenue source in the current treatment framework. The market for Fanconi Anemia is expected to experience positive growth with the approval of potential drugs like LV RP-L102, FP-045, and others.  In 2023, the overall market value of Fanconi Anemia across the 7MM was approximately USD 51 million. Forecasts suggest significant expansion in the market size throughout the projected period.

 

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Fanconi Anemia Market Strengths

• Fanconi anemia is a rare disease; thus, companies developing treatment options for the same can possess several advantages like market exclusivities, premium pricing, subsidy for conducting trials, and several other benefits from the government bodies for R&D.

 

Fanconi Anemia Market Opportunities

• There is a huge opportunity for companies coming up with therapies as there will be fewer competition in the market. If the drugs are launched they will be in huge demand keeping in mind the unmet needs for this indication.

 

Scope of the Fanconi Anemia Market Report

• Study Period: 2020–2034
• Coverage: 7MM
• Fanconi Anemia Companies: Rocket Pharma, Foresee Pharmaceuticals, Jasper Therapeutics, Novartis, and others
• Fanconi Anemia Therapies: RP-L102, FP-045, PS191, Deferasirox, and others
• Fanconi Anemia Therapeutic Assessment: Fanconi Anemia current marketed and Fanconi Anemia emerging therapies
• Fanconi Anemia Market Dynamics: Fanconi Anemia market drivers and Fanconi Anemia market barriers 
• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
• Fanconi Anemia Unmet Needs, KOL’s views, Analyst’s views, Fanconi Anemia Market Access and Reimbursement 

 

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Table of Contents 

1. Fanconi Anemia Market Report Introduction

2. Executive Summary for Fanconi Anemia

3. SWOT analysis of Fanconi Anemia

4. Fanconi Anemia Patient Share (%) Overview at a Glance

5. Fanconi Anemia Market Overview at a Glance

6. Fanconi Anemia Disease Background and Overview

7. Fanconi Anemia Epidemiology and Patient Population

8. Country-Specific Patient Population of Fanconi Anemia 

9. Fanconi Anemia Current Treatment and Medical Practices

10. Fanconi Anemia Unmet Needs

11. Fanconi Anemia Emerging Therapies

12. Fanconi Anemia Market Outlook

13. Country-Wise Fanconi Anemia Market Analysis (2020–2034)

14. Fanconi Anemia Market Access and Reimbursement of Therapies

15. Fanconi Anemia Market Drivers

16. Fanconi Anemia Market Barriers

17.  Fanconi Anemia Appendix

18. Fanconi Anemia Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

 

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