Top Primary Immunodeficiency Therapeutics Companies

Skyquest Technology's expert advisors have carried out comprehensive research and identified these companies as industry leaders in the Primary Immunodeficiency Therapeutics Market. This Analysis is based on comprehensive primary and secondary research on the corporate strategies, financial and operational performance, product portfolio, market share and brand analysis of all the leading Primary Immunodeficiency Therapeutics industry players.

Primary Immunodeficiency Therapeutics Market Competitive Landscape

The primary immunodeficiency (PI) therapeutics industry is highly concentrated, with a few multinational drug companies controlling a large majority of the market. Multinational distribution networks and large-scale plasma collection networks are some of the assets these players use. The terrain is changing, though, due to the increased competition that is building in the area of gene therapy. Small, focused biotechnology companies are entering the later phases of clinical trials with the aim of creating therapies that are curative. This type of diversity is working to drive innovation and enhance the long-term treatment modalities available to individuals with PI worldwide.

  • Orchard Therapeutics: Formed in 2015 and headquartered in the United Kingdom, Orchard Therapeutics aims to advance gene therapies for the treatment of rare genetic diseases like primary immunodeficiencies. Consequently, it came up with Strimvelis, the first ex vivo gene therapy for ADA-SCID to be approved in Europe. Despite this, the company remains committed to working on pipeline projects for other severe PIs. Through their endeavors, Orchard has succeeded in broadening the scope of potentially curative treatments with only a one-time dose and proved actual-world success in shifting the landscape of primary immunodeficiency therapeutics.
  • Mustang Bio: In 2015, Mustang Bio, a United States-based biotechnology company, started focusing on cell and gene treatments for cancer and rare disorders. A gene therapy called MB-107 is currently in clinical development for the treatment of X-linked severe combined immunodeficiency (X-SCID). This gene therapy is regarded as its closest rival. Mustang Bio has a unique manufacturing platform and collaborates with some of the world's most renowned academic institutions. The company has an impact on the primary immunodeficiency therapeutics market by addressing unmet medical needs with novel genetically based therapies that are potentially curative.

Top Player’s Company Profile

  • CSL Behring
  • Takeda Pharmaceutical
  • Grifols
  • Kedrion Biopharma
  • Octapharma
  • ADMA Biologics
  • Pfizer
  • Orchard Therapeutics
  • Rocket Pharmaceuticals
  • Bluebird Bio
  • LFB Group
  • Bio Products Laboratory (BPL)
  • China Biologic Products Holdings
  • Biotest AG
  • GC Pharma (Green Cross Corporation)

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Global Primary Immunodeficiency Therapeutics Market size was valued at USD 2.96 Billion in 2024 and is poised to grow from USD 3.13 Billion in 2025 to USD 4.87 Billion by 2033, growing at a CAGR of 5.7% during the forecast period (2026–2033). 

The primary immunodeficiency (PI) therapeutics industry is highly concentrated, with a few multinational drug companies controlling a large majority of the market. Multinational distribution networks and large-scale plasma collection networks are some of the assets these players use. The terrain is changing, though, due to the increased competition that is building in the area of gene therapy. Small, focused biotechnology companies are entering the later phases of clinical trials with the aim of creating therapies that are curative. This type of diversity is working to drive innovation and enhance the long-term treatment modalities available to individuals with PI worldwide. 'CSL Behring (USA) ', 'Grifols (Spain) ', 'Octapharma (Switzerland) ', 'Takeda Pharmaceutical Company Limited (Japan) ', 'Biotest AG (Germany) ', 'ADMA Biologics (USA) ', 'Bio Products Laboratory (BPL) (USA) ', 'LFB SA (France) ', 'Kedrion Biopharma S.p.A. (Italy) ', 'Pfizer Inc. (USA) ', 'Orchard Therapeutics (UK) ', 'Rocket Pharmaceuticals (USA) ', 'Bluebird Bio (USA) ', 'China Biologic Products Holdings (China) ', 'GC Pharma (South Korea) ', 'Baxter International Inc. (USA) ', 'X4 Pharmaceuticals (USA) ', 'Novartis AG (Switzerland) ', 'Sanofi S.A. (France) ', 'Bristol Myers Squibb (USA)'

Advancements in diagnostic technology, specifically over the last several years next-generation sequencing (NGS) and genetic screening, have played an important role in minimizing the period required for a diagnosis of primary immunodeficiency (PI) disease. An early diagnosis not only is it feasible to initiate treatment at an earlier date, but also therapeutic results become better because they minimize the burden of infection. This type of increased diagnostic accuracy results in enhanced PI management, greater compliance, and better patient quality of life, all resulting in a higher market demand for PI medication.

Individualized Immunoglobulin Dosing for Patients: Dose algorithms derived from artificial intelligence are helping to revolutionize PI treatment by allowing for the possibility of individualizing immunoglobulin dose recommendations. Patient weight, frequency of infections, and metabolic rate are all parameters that are considered in an effort to optimize treatment regimens and prevent side effects. This strategy enhances treatment adherence, reduces the likelihood of under- or overdosing, and optimizes use of available resources. To date, clinical trials are ongoing to confirm the efficacy of artificial intelligence (AI) in various patient populations. The early results suggest that AI needs to be employed for a more extensive duration in the clinical environment.

High insurance coverage, early diagnosis, and strongly approved therapy pipeline in North America have resulted in the region's leadership of the primary immunodeficiency therapeutics sector. International pharmaceutical corporations with a United States headquarters are leaders in research in gene and immunoglobulin therapies. An ongoing surge in demand has been induced as a consequence of mounting identification rates, which have been induced by activist organizations like the Immune Deficiency Foundation (IDF). The efforts have led to wider registries of patients and national education campaigns.

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Global Primary Immunodeficiency Therapeutics Market
Primary Immunodeficiency Therapeutics Market

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