Global Fanconi Anemia Market - 2023-2030

Overview

Fanconi anemia is a rare hereditary disorder that typically manifests in children with inadequate blood cell production at relatively young ages and is characterized by bone marrow failure and cancer propensity. Hematopoietic stem cell transplantation from a healthy donor, in addition to other palliative treatments, is currently the preferred course of action for these patients.

Even though this kind of transplant has undergone significant advancements recently, not all patients have a suitable donor. Additionally, pre-transplant chemotherapy is necessary for this procedure, and rejection issues as well as other long-term side effects are possible.

Dynamics

Active Major Players

In May 2022, the Center for Definitive and Curative Medicine (CDCM) at the Stanford University School of Medicine is conducting research using JPS191 as a conditioning agent to treat Fanconi anemia under the sponsorship of Jasper Therapeutics, Inc., a biotechnology company that specializes in hematopoietic cell transplant therapies. The company announced the dosing of the first patient in this study.

Stanford Medicine is conducting a Phase 1/2 clinical trial (NCT04784052) using JSP191 to treat patients with Fanconi anemia who have bone marrow failure and need an allogeneic transplant from a non-sibling donor in compliance with the sponsored research agreement.

Increasing Research Activities

Despite the fact that hematopoietic stem cell transplant normally has a survival rate of over 80%, the therapy is linked to substantial side effects, such as 100-day mortality and an elevated risk of malignancies. Researchers are testing a gene treatment that uses autologous Fanconi anemia Group A CD34 + enriched hematopoietic stem and progenitor cells that engraft without the need for antecedent conditioning to address this. Phase 1/2 clinical studies for the Rocket Pharmaceuticals-created medication, RP-L102, are now underway.

To evaluate phenotypic correction, engraftment, clinical response, and safety of RP-L102, 12 patients at least 1 year of age with no HLA-matched sibling donor have been treated and monitored as of the data cutoff in October 2022. The absence of conditioning allowed for the sustained increase in bone marrow colony-forming cell (BM CFC MMC) resistance, concurrent genetic correction, and hematologic stabilization that the researchers saw in at least six out of ten patients with at least a year of follow-up.

Increasing Treatment Options

Blood and bone marrow stem cell transplant: FA patients with bone marrow failure typically have blood and bone marrow stem cell transplants as part of their treatment. It is the sole therapy that could reverse FA's blood-related complications. Transfusion of blood: While not a curative procedure, blood transfusions can offer supportive care.

Androgen therapy: The use of synthetic male hormones occasionally increasing the number of red blood cells and platelets while improving stem cell growth, Trusted Source is supportive rather than curative. Blood and bone marrow stem cell transplant: FA patients with bone marrow failure typically have blood and bone marrow stem cell transplants as part of their treatment. It is the sole therapy that could reverse FA's blood-related complications.

Technological advances

For the restoration of hematological function and the prevention of MDS/AML formation in FA patients, new, potentially curative DNA-directed gene treatments are being created thanks to technological advancements. The genetic mutations that cause FA are intended to be fixed using gene therapy. Researchers seek to restore the proper operation of hematopoietic stem cells (HSCs), which are the precursors of blood cells, by inserting a functional copy of the mutant gene into the patient's cells.

In addititon, autologous stem cell transplantation, the patient's own bone marrow or blood stem cells are taken, the genetic error is fixed in a lab, and the corrected cells are then transplanted back into the patient. This might result in the creation of healthy blood cells and stop the growth of MDS/AML.

Rare Disease Challenges

Due to their rarity, rare diseases are sometimes poorly understood by the medical community. Healthcare personnel might not be familiar with the precise symptoms and traits of certain illnesses, which might result in delayed or incorrect diagnoses. Accurate diagnosis of these disorders might be difficult due to their rarity. Before acquiring a correct diagnosis, patients may go through a protracted diagnostic odyssey that involves seeing numerous doctors.

The results for patients may suffer significantly as a result of this delay. Due to the rarity of rare diseases, it is challenging to conduct extensive clinical studies and obtain the necessary information to prove a treatment's effectiveness. This may make it difficult for novel therapies to receive regulatory approval.

Drug Development Challenges

Rare disease research may be spread out over various institutions and nations, resulting in fragmented data and insufficient collaboration. For the purpose of improving research and creating therapies, sharing data and findings can be essential. Inadequate funding for research and treatment development may be the result of low public awareness of and advocacy for uncommon diseases. This may impede efforts to uncover the underlying causes of these illnesses and create novel treatments.

It can take a long time to conduct thorough research and create new therapeutics, including gene therapies and stem cell therapies. Access to potentially life-saving therapies for patients may be impacted by development delays.

Segment Analysis

The global fanconi anemia market is segmented based on drug type, age group, end-user and region.

Hematopoietic Growth Factors Accounted for Dominant Market share during the Forecast period

In the bone marrow, hematopoietic growth factors (HGFs) are chemicals that promote the development of blood cells. To support and improve the development of particular blood cell types, they are frequently employed in medical practice. Hematopoietic growth factors can be used as part of the overall care plan for Fanconi Anemia (FA), a rare hereditary condition that compromises bone marrow function.

A hematopoietic growth factor called erythropoietin (EPO) promotes erythropoiesis or the creation of red blood cells. Anemia can occur in patients with Fanconi Anemia because red blood cell synthesis is compromised. To treat this anemia and enhance the patient's general level of energy and quality of life, EPO therapy may be recommended.

Geographical Penetration

Increasing Research Activities in North America

North America has been a dominant force in the global fanconi anemia market. Fanconi anemia is an extremely rare disease. About 31 children are born with FA per year in the United States, according to the Fanconi Anemia Research Fund. The incidence rate, or the likelihood of a child being born with FA, is about 1 in 136,000 births.

Researchers estimate that the incidence of people carrying an abnormality in an FA gene in the United States is 1 in 181. Research, information exchange, and the creation of viable Fanconi Anemia remedies have all been made possible through partnerships between academic institutions, healthcare facilities, pharmaceutical firms, and patient advocacy organizations.

Technologies and advances in gene therapy have shown promise in the treatment of genetic diseases like Fanconi anemia. Gene therapy research and development are concentrated in North America, the region which may have an impact on FA treatment options.

COVID-19 Impact Analysis

The outbreak of the COVID-19 pandemic in late 2019 created unprecedented challenges for industries worldwide, including the global fanconi anemia market. Patients with fanconi anemia frequently have impaired immunological function and dysfunctional bone marrow. They may be more susceptible as a result to serious respiratory infections like COVID-19. Therefore, strong attention to preventive measures, such as social isolation, mask use, and proper hand cleanliness, is essential to lower the chance of viral exposure.

FA patients must undergo routine checkups and medical appointments in order to maintain good health. However, there may have been delays in healthcare services during the pandemic, including as canceled appointments, fewer in-person visits, and a switch to telemedicine consultations. To ensure continuing care and necessary modifications to treatment plans, patients and their carers should stay in touch with their healthcare providers.

By Drug Type

• Hematopoietic Growth Factors
• Gene Therapy
• Androgen Therapy
• Others

By Age Group

• Pediatric
• Adults

By End-users

• Clinics
• Hospitals
• Others

By Region

• North America
  • U.S.
  • Canada
  • Mexico

• Europe
  • Germany
  • UK
  • France
  • Italy
  • Spain
  • Rest of Europe

• South America
  • Brazil
  • Argentina
  • Rest of South America

• Asia-Pacific
  • China
  • India
  • Japan
  • Australia
  • Rest of Asia-Pacific

• Middle East and Africa

Competitive Landscape

The major global players in the market include: Rocket Pharmaceuticals, Forty-Seven, Inc., Jasper Therapeutics, Inc., Genethon, Abeona Therapeutics Inc., and others.

Why Purchase the Report?

• To visualize the global fanconi anemia market segmentation based on drug type, age group, end-user and region as well as understand key commercial assets and players.
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The global fanconi anemia market report would provide approximately 61 tables, 58 figures and 186 Pages.

Target Audience 2023

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies