Global Leukocyte Adhesion Deficiency (LAD-I) Market Analysis 2022-2031

Leukocyte Adhesion Deficiency (LAD-I) Market by Treatment (Hematopoietic Stem Cell Transplantation, Recombinant Human Interferon-gamma Treatment, Prophylactic Immunoglobulin Therapy, Antimicrobial Therapy, Prophylactic Therapy, Fucose Supplementation, Monoclonal Antibodies, Coagulation Factors), by End Users (Hospitals, Specialty Clinics and Others: Global Opportunity Analysis and Industry Forecast, 2022-2031

The Leukocyte Adhesion Deficiency (LAD-I) market was valued at $7,500 million in 2022. It is projected to grow at a CAGR of 6.2% from 2023 to 2031 and reach more than 13,000 million by the end of 2031.

Analysts’ Viewpoint by Cognate Lifesciences

Leukocyte Adhesion Deficiency (LAD-I) is a rare genetic disorder that affects the immune system's ability to fight infections. Currently, there is no cure for LAD-I, and treatment options are limited. However, there is a growing interest in developing new therapies to address this unmet medical need. Analysts view the LAD-I market as small but potentially lucrative, given the high unmet need and the rarity of the disease. The market is expected to grow steadily in the coming years, driven by an increasing number of diagnosed cases and the development of novel therapies.

Currently, there are several companies involved in the development of treatments for LAD-I, including gene therapies and biologics. These companies are likely to face challenges in bringing their products to market due to the high cost of development and the limited patient population. However, with the growing interest in rare diseases and the increasing number of incentives offered by regulatory bodies, there is optimism that new therapies for LAD-I will be approved in the near future. As such, the LAD-I market presents an attractive opportunity for companies that are willing to invest in this area.

Leukocyte Adhesion Deficiency (LAD-I) Overview 

Leukocyte Adhesion Deficiency (LAD-I) is a rare genetic disorder that affects the immune system's ability to fight infections. It is caused by mutations in the ITGB2 gene, which encodes for the beta-2 integrin protein, a molecule that is essential for the attachment of white blood cells (leukocytes) to blood vessel walls and the subsequent migration of these cells to sites of infection. This genetic disorder affects the proper functioning of white blood cells, resulting in recurrent bacterial infections, impaired wound healing, and chronic inflammation.

One of the primary drivers of the LAD-I market is the high unmet medical need. Currently, there are very less effective treatments available for LAD-I, which creates a significant opportunity for companies to develop and market novel therapies. Additionally, the rarity of the disease means that there are few competing therapies, which further increases the opportunity for companies. Regulatory bodies such as the US FDA and the EMA offer incentives to encourage the development of treatments for rare diseases like LAD-I. These incentives help to expedite the review and approval process of drugs and treatments, as well as provide tax credits and exclusivity periods for approved treatments. Here are some examples of these regulatory incentives in action:

The FDA offers several incentives for the development of treatments for rare diseases, including LAD-I. One such incentive is the Breakthrough Therapy Designation, which is designed to expedite the development and review of drugs for serious or life-threatening conditions. In 2019, Orchard Therapeutics received Breakthrough Therapy Designation from the FDA for its investigational gene therapy OTL-103, which aims to restore the function of the beta-2 integrin protein in people with LAD-I. This designation allowed Orchard Therapeutics to expedite the development and review of the therapy, which could potentially benefit patients with LAD-I.

The EMA also offers several regulatory incentives for rare disease treatments. One such incentive is the Orphan Designation, which provides a range of benefits to companies developing treatments for rare diseases. These benefits include fee reductions for regulatory procedures, access to scientific advice, and ten years of market exclusivity in the European Union. In 2021, the EMA granted Orphan Designation to the gene therapy for LAD-I developed by Cognate Bioservices in partnership with the National Institutes of Health. This designation provides regulatory and financial support to Cognate Bioservices to advance the development of the therapy. These incentives are essential in encouraging companies to invest in the development of treatments for rare diseases, which can significantly improve the lives of patients suffering from these conditions.

Moreover, gene therapy is a treatment approach that involves the insertion of a functional copy of a faulty gene into a patient's cells to correct a genetic disorder. In the case of LAD-I, the faulty gene is ITGB2, which encodes the beta-2 integrin protein that is essential for white blood cells to adhere to blood vessel walls and fight infections. In individuals with LAD-I, this protein is either absent or nonfunctional, leading to recurrent bacterial and fungal infections.

Orchard Therapeutics, a biotechnology company, has developed an investigational gene therapy called OTL-103 for the treatment of LAD-I. This therapy involves the extraction of a patient's bone marrow stem cells, which are then genetically modified to introduce a functional copy of the ITGB2 gene. The modified stem cells are then infused back into the patient's bloodstream, where they migrate to the bone marrow and start producing healthy white blood cells with functional beta-2 integrin protein.

New product launches to flourish in the market

In April 2022, Pharming Group N.V. presented positive data from the pivotal Phase II/III trial of leniolisib for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a primary immunodeficiency. The study met its primary endpoint, demonstrating a statistically significant reduction in the rate of moderate to severe infections in patients treated with leniolisib compared to placebo. This could potentially lead to leniolisib becoming a new treatment option for patients with APDS. In September 2022, Lactiga Therapeutics raised USD 1.6 million in oversubscribed pre-seed financing for developing therapeutics for patients with primary immunodeficiency diseases. The company's lead program is focused on developing a first-in-class small molecule therapy for the treatment of common variable immunodeficiency (CVID), another type of PID.

Segment Overview:

By Treatment: The Leukocyte Adhesion Deficiency (LAD-I) market is divided into Hematopoietic Stem Cell Transplantation, Recombinant Human Interferon-gamma Treatment, Prophylactic Immunoglobulin Therapy, Antimicrobial Therapy, Prophylactic Therapy, Fucose Supplementation, Monoclonal Antibodies and Coagulation Factors. These treatments aim to address the underlying cause of LAD-I, which is a deficiency in the beta-2 integrin protein, leading to impaired immune function. While there is no cure for LAD-I, these treatments can help manage the symptoms of the disease and reduce the risk of complications such as infections. The availability and effectiveness of these treatments may vary, and research is ongoing to develop more effective therapies for LAD-I.

By End-user: The Leukocyte Adhesion Deficiency (LAD-I) market is segmented by end-user into hospitals, specialty clinics, and others. Hospitals and specialty clinics are the primary end-users for LAD-I treatment, as these facilities typically have the resources and expertise necessary to manage rare and complex diseases. In addition, they may have access to specialized diagnostic and therapeutic equipment and may be able to provide comprehensive care to patients with LAD-I. Other end-users may include research institutions and academic medical centers that are involved in the development and testing of new therapies for LAD-I, as well as government agencies and non-profit organizations that support research and advocacy for rare diseases.

By Region:

The North American Leukocyte Adhesion Deficiency (LAD-I) market is one of the largest in the world, driven by factors such as increasing disease awareness, regulatory incentives, and significant investment in research and development. The United States is a major contributor to the market, with a high prevalence of LAD-I cases and a strong presence of biotechnology and pharmaceutical companies that are developing novel therapies. For example, Orchard Therapeutics, a biotechnology company based in the United States, received breakthrough therapy designation from the FDA for its investigational gene therapy OTL-103, which aims to treat LAD-I. Other companies such as Cognate Bioservices are also developing novel treatments for LAD-I in North America.

The Asia-Pacific Leukocyte Adhesion Deficiency (LAD-I) market is a growing market, driven by factors such as increasing awareness of rare diseases, improving healthcare infrastructure, and rising investment in research and development. Japan and China are the major contributors to the market due to their large population and increasing healthcare expenditure. For example, in Japan, Stemirna Therapeutics Co. Ltd. has developed a novel gene therapy for LAD-I and received approval for conducting clinical trials. Additionally, Indian biotech company, XyloCor Therapeutics, has also developed a gene therapy for LAD-I and received orphan drug designation from the FDA. As healthcare infrastructure continues to improve in the region, the market for LAD-I treatments is expected to grow.

Competitive analysis and profiles of the major players in the Leukocyte Adhesion Deficiency (LAD-I) market, such as Orpha Labs, Avalo Therapeutics, Inc., Rocket Pharmaceuticals Inc., Sigma-Aldrich, Aspen Neuroscience, Magenta Therapeutics, Rubius Therapeutics, Enochian Biosciences, Sana Biotechnology and Vertex Pharmaceuticals. Major players have adopted product launch and acquisition as key developmental strategies to improve the product portfolio of the Leukocyte Adhesion Deficiency (LAD-I) market.    

Market Scope and Structure Analysis

Key Segments Covered

Type Treatment

• Hematopoietic Stem Cell Transplantation
• Recombinant Human Interferon-gamma Treatment
• Prophylactic Immunoglobulin Therapy
• Antimicrobial Therapy
• Prophylactic Therapy
• Fucose Supplementation
• Monoclonal Antibodies
• Coagulation Factors

End-user

• Hospitals
• Specialty Clinics
• Others

Region

• North America
  • U.S.
  • Canada
  • Mexico
• Europe
  • Germany
  • UK
  • France
  • Italy
  • Spain
  • Rest of Europe
• Asia-Pacific
  • China
  • Japan
  • South Korea
  • India
  • Australia
  • Rest of Asia-Pacific
• LAMEA
  • Latin America
  • Middle East
  • Africa