What is the global market size of Orphan Drug Market?

Global Orphan Drug Market size was valued at USD 87.44 billion in 2021 and is poised to grow from USD 174.11 billion in 2022 to USD 359.86 billion by 2030, growing at a CAGR of 9.5% in the forecast period (2023-2030).

Who are the key vendors in the Global Orphan Drug Market?

The Global Orphan Drug Market is relatively fragmented, with a high level of competition. The prominent players operating in the orphan drug market are constantly 'Vertex Pharmaceuticals', 'Alexion Pharmaceuticals', 'BioMarin Pharmaceutical', 'Shire (now part of Takeda)', 'Sanofi Genzyme', 'Celgene (now part of Bristol Myers Squibb)', 'Genentech (a subsidiary of Roche)', 'Pfizer', 'Novartis', 'Johnson & Johnson', 'AbbVie', 'Amgen', 'Ultragenyx Pharmaceutical', 'CSL Behring', 'Merck & Co.', 'Grifols', 'Sobi (Swedish Orphan Biovitrum)', 'Ipsen', 'Jazz Pharmaceuticals', 'AstraZeneca'

Orphan Drugs Market Size, Share, Growth Analysis, By Drug Type (Biological, Non-biological), By Disease Type (Oncology, Hematology), By Distribution Channel (Pharmacy, Hospital), By Region - Industry Forecast 2025-2032

Report ID: SQMIG30A2005 | Region: Global | Published Date: November, 2024
Pages: 211 | Tables: 65 | Figures: 75

Orphan Drug Market News

In April 2022, according to news, the product's creator Shanghai Henlius Biotech, the FDA has qualified serplulimab, also known as Hansizhuang, as an orphan medication for the treatment of small-cell lung cancer.
In March 2022, ABIONYX Pharma reported that the USFDA had granted Bio-HDL CER-001 orphan-drug designation (ODD) for the treatment of lecithin-cholesterol acyltransferase deficiency, also known as LCAT deficiency.

Orphan Drug Market Report Snapshots

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Global Orphan Drugs Market size was valued at USD 175.06 billion in 2022 and is poised to grow from USD 195.76 billion in 2023 to USD 478.49 billion by 2031, growing at a CAGR of 11.82% during the forecast period (2024-2031).

The Global Orphan Drug Market is relatively fragmented, with a high level of competition. The prominent players operating in the orphan drug market are constantly 'Novartis AG ', 'Pfizer Inc. ', 'Sanofi ', 'Bristol-Myers Squibb Company ', 'GSK plc ', 'AbbVie Inc. ', 'Merck & Co., Inc. ', 'Johnson & Johnson Private Limited ', 'F. Hoffmann-La Roche Ltd ', 'Vertex Pharmaceuticals ', 'BioMarin Pharmaceutical ', 'Takeda Pharmaceutical Company Limited ', 'Roche ', 'Amgen ', 'Ultragenyx Pharmaceutical ', 'CSL Behring ', 'Grifols ', 'Sobi (Swedish Orphan Biovitrum) ', 'Ipsen ', 'Jazz Pharmaceuticals ', 'AstraZeneca'

The development of orphan medications under research has been significantly influenced by the development of new technologies. Due recent advancements in genomics and the wider application of gene sequencing, made it possible in case of rare disease. furthermore, additional technologies like gene therapy and antisense therapy have made it feasible for us to target certain disorders in ways that were not previously possible. Combined, technological advancements have made it easier to both diagnose and cure uncommon genetic disorders, which has made them more manageable for pharmaceutical companies.

Since companies employ far lower criteria for showing treatment effectiveness, the expansion of the use of auxiliary or surrogate endpoints within clinical trials for orphan pharmaceuticals has significantly reduced the time and cost needed to undertake R&D. This can accelerate the growth of the Orphan Drug Market.

In terms of CAGR, North America is expected to grow quickly in the years to come. This is attributed to the increase in patients in this area who are susceptible to rare diseases. The unhealthy lifestyle and stressful lifestyle are the key factors that cause this disease to spread in this area. In order to reduce the number of patients brought on by this disease, government organisations like NGO are investing a significant amount of money in this region.

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Orphan Drugs Market Size, Share, Growth Analysis, By Drug Type (Biological, Non-biological), By Disease Type (Oncology, Hematology), By Distribution Channel (Pharmacy, Hospital), By Region - Industry Forecast 2025-2032

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