Rare Disease Treatment Market - Top Companies and Manufacturers

Companies Dominating the Rare Disease Treatment Landscape

Companies in the rare disease treatment market are majorly focusing on securing U.S. FDA orphan drug approvals for benefits such as tax credits and market exclusivity. In September 2024, UCB received U.S. FDA approval for BIMZELX for treating adults with active psoriatic arthritis (PsA) active non-radiographic axSpA with objective signs of inflammation, and active ankylosing spondylitis. They also pursue partnerships and acquisitions to broaden their pipelines and invest heavily in R&D, especially in gene and cell therapies. Furthermore, collaborations with patient advocacy groups help raise awareness and tailor treatments to meet specific patient needs.

AbbVie Inc.
- Company Overview
- Business Strategy
- Key Product Offerings
- Financial Performance
- Key Performance Indicators
- Risk Analysis
- Recent Development
- Regional Presence
- SWOT Analysis
Alexion Pharmaceuticals, Inc.
Amgen Inc
AstraZeneca PLC
Baxter International
Bayer AG
Bristol-Myers Squibb
Eli Lilly and Company
F. Hoffmann La Roche Ltd
GSK plc
Johnson & Johnson
Merck & Co. Inc.
Pharmacyclics LLC
Seagen Inc.
Takeda Pharmaceutical Company Limited.
Vertex Pharmaceutical, Inc.

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Rare Disease Treatment Market Size & Share, Growth Analysis 2037

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In September 2024, Chugai Pharmaceutical Co., Ltd. received approval from the MHLW Evrysdi Dry Syrup 60 mg (risdiplam) for an additional indication of pre-symptomatic SMA.
In December 2023, Novartis received U.S. FDA approval for Fabhalta(iptacopan), which is a Factor B inhibitor. It is the first oral monotherapy for the treatment of adults with PNH.

Frequently Asked Questions (FAQ)

In the year 2024, the industry size of the rare disease treatment market was over USD 224.6 billion.

The market size for the rare disease treatment market is projected to reach USD 652.9 billion by the end of 2037 expanding at a CAGR of 9.3% during the forecast period i.e., between 2025-2037.

The major players in the market are Bayer AG, Bristol-Myers Squibb, Eli Lilly and Company, F. Hoffmann La Roche Ltd, and others.

In terms of route of administration segment, the oral segment is anticipated to garner the largest market share by 2037 and display lucrative growth opportunities during 2025-2037.

The market in North America is projected to hold the largest market share of 45.5% by the end of 2037 and provide more business opportunities in the future.

Rare Disease Treatment Market - Top Companies and Manufacturers

Companies Dominating the Rare Disease Treatment Landscape

Browse Key Market Insights with Data Illustration:

In the News

Frequently Asked Questions (FAQ)

How big is the rare disease treatment market?

What is the outlook for the rare disease treatment market?

Who are the major players dominating the rare disease treatment market?

Which route of administration segment will capture the significant market share for the rare disease treatment market by 2037?

Which region will provide more business opportunities for the growth of the rare disease treatment market in the future?