US Orphan Drugs Market Surpasses USD 150 Billion Opportunity by 2028 – Key Players Amgen, Horizon Therapeutics, and Alexion at the Forefront – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “US Orphan Drugs Market, Drugs Sales, Price, Dosage & Clinical Trials Insight 2028” report has been added to ResearchAndMarkets.com’s offering.

Explore the Global Advanced High Strength Steel Market, estimated at USD 28.13 Bn in 2023 and projected to reach USD 50.96 Bn by 2028, with a steady growth rate of 12.62%. This comprehensive report analyzes industry dynamics, key players, and strategies to help businesses strengthen their market foothold.

Report Highlights:

• US Orphan Drugs Market Opportunity: > USD 150 Billion By 2028
• Orphan Drugs Dosage, Price and Treatment Cost: > 350 Orphan Drug
• Annual and Quarterly Sales Insight (2019 – Q1’2023):> 100 Orphan Drugs
• US Orphan Drugs Reimbursement Scenario: Medicare, Medicaid, Private Insurers
• Active Clinical Trials Insight By Company, Indication and Phase: 1000 Orphan Drugs
• Marketed Orphan Drugs Clinical Insight By Company and Indication: > 400 Orphan Drugs
• Competitive Landscape: 75 Companies

A selection of companies mentioned in this report includes

• AbbVie
• Alexion (AstraZeneca Rare Disease Group)
• Alnylam Pharmaceuticals
• Amgen
• Amicus Therapeutics
• Array BioPharma
• Astellas Pharma
• AstraZeneca
• Bayer HealthCare
• Beacon Therapeutics
• BioMarin Pharmaceutical
• Bioverativ
• Boehringer Ingelheim
• Bristol-Myers Squibb
• Celgene Corporation
• Chiesi
• Chugai Pharmaceutical
• Collaborations Pharmaceuticals
• CSL
• Daiichi Sankyo Company
• Eisai Co Ltd
• Eli Lilly & Company
• Emergent BioSolutions
• Ferring Pharmaceuticals
• Fred Hutchinson Cancer Research Center
• Flavocure Biotech
• Genentech
• Genmab
• Gilead Sciences

Considering the increasing burden of patients suffering from rare and orphan diseases, the US FDA has taken several steps to increase the interest of the research and pharmaceutical companies to these forgotten diseases. One of the fundamental actions taken by the FDA is the assignment of the orphan drug designation, which gives developers benefits in the course of the research and development, clinical assessment, and marketing of the drug.

The orphan drug designation has been granted to over 6500 drugs and compounds, and around 100 of these have received regulatory approvals. Therefore, though the number is less, these drugs have been essential for improving the treatment outcomes of patients suffering from rare orphan diseases. Taking into account the continued success of several drugs in the US pharmaceutical market, it is evident these therapeutic products are faring well in spite of the small patient base, which makes it an interesting domain to explore.

Earlier rare diseases were neglected due to low profitability feasibility, but the enactment of FDA’s Orphan Drug Act 1983, succeeding regulatory reforms, government funding support, and extensive research by pharmaceutical companies led to exponential growth in the US orphan drugs market. These favorable parameters resulted in more than 1000 orphan-designated drugs in clinical trials and more than 400 orphan-designated drugs being commercially available in the market.

This number is further expected to surpass 500 orphan-designated drugs in the market by 2028 driven by the increasing focus of pharmaceutical companies on rare diseases.

Many drugs that were given the orphan designation for rare indications have already gained US FDA approval for a different, non-orphan indication. Observation studies, anecdotal evidence, or unexpected clinical results from clinical trials involving patients with unrelated indications are what lead researchers to the conclusion that the same drug may also be utilized for a rare indication.

As a result, it frequently happens that pharmaceutical companies are unaware of the potential of their products as therapeutic treatments for rare diseases. On the plus side, completing clinical trials for uncommon diseases, and subsequently being given the orphan drug designation, can aid in growing the patient base for the company and the drug, which will later boost revenues.

For instance, Eylea, which was initially approved in 2011 for the treatment of wet age-related macular degeneration, was given its first orphan drug designation for the treatment of retinopathy of prematurity in February 2023, and was immediately approved for the same indication. Eight approvals for Eylea have been received since 2011 before this recent new approval, which supports the fact that many pharmaceutical companies are unable to foresee the potential of their products in orphan indications. This only emphasizes the importance of conducting clinical trials for orphan indications that have underlying characteristics similar to more prevalent diseases.

It has been observed that the majority of orphan drugs were developed by modest-scale biotechnology firms and then acquired by prominent pharmaceutical companies. When it comes to exploring understudied diseases and disorders, smaller biotechnology companies have some distinct benefits. Their founders often have better knowledge and experience conducting research and developing medicines for rare diseases.

Key Topics Covered:

1. US Orphan Drugs Market Overview

2. Partnerships & Collaborations Concerning Orphan Drugs

3. US Orphan Drugs Market Trends By Indication

4. US Orphan Drugs Reimbursement Scenario

5. US Orphan Designated Drugs Dosage, Price & Treatment Cost Insight

6. US Orphan Designated Drugs Sales Insight (2019 – Q1’2023)

7. US Orphan Designated Drugs Clinical Trials Insight

8. US Orphan Drugs Clinical Trials By Company, Indication & Phase

9. Marketed Orphan Drugs Clinical Insight By Company & Indication

10. Competitive Landscape

For more information about this report visit https://www.researchandmarkets.com/r/msdrn7

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