The global orphan drugs market is anticipated to grow at a CAGR of 11.8% during the forecast period (2023-2030). Enzyme replacement therapy (ERT), gene therapy, small molecule drugs, biologics, and stem cell therapy are type of treatments used to cure rare diseases.
The market’s growth is attributed to advancements in biotechnology and personalized medicine across the globe. The development of targeted medicines has become possible owing to the developments in biotechnology, genetics, and precision medicine. The advancements can be particularly significant for treating rare disorders, where a one-size-fits-all strategy can prove to be efficient. According to the National Institute of Health (NIH.gov), in February 2023, NMOSD is a rare chronic, autoimmune-mediated disease that affects the nervous system, primarily damaging the optic nerves and the spinal cord. It is estimated to affect 0.37 to 10 people per 100,000, with a female predominance and a median age of onset of 32 to 41 years. The diagnosis includes clinical criteria and MRI findings, as well as the presence of antibodies against the aquaporin-4 water channel (AQP4). If the serological testing is unavailable or the antigens for AQP4 are negative, more extensive clinical and imagistic tests may be needed. Generally, the disability resulting from NMOSD is caused by severe demyelination and extensive axonal damage. NMOSD is a pathology which involves interleukin-6. Its main symptoms are bilateral optic neuritis, transverse myelitis, pain in the legs and trunk, intractable hiccups, nausea, or vomiting, excessive somnolence or narcolepsy, reversible posterior encephalopathy syndrome, hypothalamic dysfunction, autonomic manifestations, and seizures. Investigations include spinal cord MRI, brain MRI, and MRI of the orbits.
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Market Coverage
- The market number available for – 2022-2030
- Base year- 2022
- Forecast period- 2023-2030
Segment Covered-
- By Drug Type
- By Disease Type
- By Distribution Channel
Regions Covered-
- North America
- Europe
- Asia-Pacific
- Rest of the World
Competitive Landscape- includes AstraZeneca., At F. Hoffmann-La Roche Ltd, Bayer AG, Bristol-Myers Squibb India Pvt. Ltd., Daiichi Sankyo Co. Ltd., and others.
Orphan Drugs Market Report Segment
By Drug Type
- Biological
- Non-biological
By Disease Type
- Oncology
- Hematology
- Neurology
- Cardiovascular
- Others (Autoimmune Diseases, and Rare Blood Disorders)
By Distribution Channel
- Hospital Pharmacy
- Retail Pharmacy
- Online Pharmacy
- Others (Specialty Pharmacies, and Direct-to-Patient Distribution)
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Orphan Drugs Market Report Segment by Region
North America
- United States
- Canada
Europe
- UK
- Germany
- Spain
- France
- Italy
- Rest of Europe
Asia-Pacific
- India
- China
- Japan
- South Korea
- Rest of APAC
Rest of the World
- Latin America
- Middle East and Africa
Company Profiles
- At F. Hoffmann-La Roche Ltd
- Bayer AG
- Bristol-Myers Squibb India Pvt. Ltd.
- Daiichi Sankyo Co. Ltd.
- GlaxoSmithKline plc
- Johnson & Johnson Pvt. Ltd.
- Novartis AG
- Pfizer Inc.
- Sanofi
- Takeda Pharmaceutical Co. Ltd.
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