Smaller Biotechnology Companies Lead Growth in the US Orphan Drugs Market - Amgen's Acquisition of Horizon Therapeutics to Expand Pipeline

DUBLIN, Aug. 4, 2023 /PRNewswire/ -- 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

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.

Additionally, these companies do not have the complex bureaucracy found in larger companies, which simplifies decision-making. In addition, they stand to lose less money if the project does not work out. Therefore, if their therapeutics are successful, the majority of these small biotechnology companies are bought by larger ones, greatly enhancing their credibility in the orphan drug market. On the same vein, larger companies are also known to purchase orphan drug candidates in exchange for royalties and milestone payments.

For instance, Amgen and Horizon Therapeutics reached a deal in December 2022 for Amgen to buy the company and add Horizon's orphan drugs Actimmune, Ravicti, Tepezza, and Uplizna to its pipeline. Although the deal has not yet been finalized, it presents Amgen with the chance to expand its assembly of orphan drugs for rare diseases.

In the past couple years, number of clinical candidates receiving the orphan drug designation have increased drastically, which can be attributed to the benefits offered by the FDA. Temferon, RAG-17, INB-400, Batiraxcept and ZYIL1 are only a few candidates that received the orphan drug designation in 2023 for a variety of rare diseases.

It is expected that as more companies understand the unmet needs of patients suffering from rare diseases, more drug candidates for these indications will enter the pipeline. The US currently leads the sale and development of orphan drugs due to the existence of numerous companies dedicated to treating rare diseases, favorable government regulations, such as Medicare/Medicaid reimbursements, and the extensive network of patient advocacy groups that work to support those with 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
7.1 By Company
7.2 By Indication
7.3 By Patient Segment
7.4 By Phase
7.5 By Priority Status

8. US Orphan Drugs Clinical Trials By Company, Indication & Phase
8.1 Research
8.2 Preclinical
8.3 Phase-I
8.4 Phase-I/II
8.5 Phase-II
8.6 Phase-II/III
8.7 Phase-III
8.8 Preregistration
8.9 Registered

9. Marketed Orphan Drugs Clinical Insight By Company & Indication

10. Competitive Landscape
10.1 AbbVie
10.2 Alexion (AstraZeneca Rare Disease Group)
10.3 Alnylam Pharmaceuticals
10.4 Amgen
10.5 Amicus Therapeutics
10.6 Array BioPharma
10.7 Astellas Pharma
10.8 AstraZeneca
10.9 Bayer HealthCare
10.10 Beacon Therapeutics
10.11 BioMarin Pharmaceutical
10.12 Bioverativ
10.13 Boehringer Ingelheim
10.14 Bristol-Myers Squibb
10.15 Celgene Corporation
10.16 Chiesi
10.17 Chugai Pharmaceutical
10.18 Collaborations Pharmaceuticals
10.19 CSL
10.20 Daiichi Sankyo Company
10.21 Eisai Co Ltd
10.22 Eli Lilly & Company
10.23 Emergent BioSolutions
10.24 Ferring Pharmaceuticals
10.25 Fred Hutchinson Cancer Research Center
10.26 Flavocure Biotech
10.27 Genentech
10.28 Genmab
10.29 Gilead Sciences
10.30 GlaxoSmithKline
10.31 GSK
10.32 Hanmi Pharmaceutical
10.33 Horizon Therapeutics plc
10.34 Incyte Corporation
10.35 Ionis Pharmaceuticals
10.36 Ipsen
10.37 Janssen Biotech
10.38 Jazz Pharmaceuticals plc
10.39 Johnson & Johnson
10.40 Kamada
10.41 Krystal Biotech
10.42 Kyowa Kirin
10.43 Leadiant Biosciences
10.44 Ligand Pharmaceuticals
10.45 Lundbeck A/S
10.46 MeiraGTx
10.47 Memorial Sloan-Kettering Cancer Center
10.48 Merck
10.49 Moderna Therapeutics
10.50 Nationwide Children's Hospital
10.51 Nonindustrial Source
10.52 Novartis
10.53 Novo Nordisk
10.54 Omeros Corporation
10.55 Onyx Pharmaceuticals (Amgen)
10.56 Otsuka Pharmaceutical
10.57 Pfizer
10.58 PTC Therapeutics
10.59 Recordati
10.60 Regeneron Pharmaceuticals
10.61 Roche
10.62 Sanofi
10.63 Servier
10.64 Shionogi
10.65 Spark Therapeutics
10.66 Sumitomo Pharma
10.67 Sutro Biopharma
10.68 Swedish Orphan Biovitrum
10.69 Takeda
10.70 Teva Pharmaceutical Industries
10.71 UCB
10.72 UniQure
10.73 Vertex Pharmaceuticals
10.74 XOMA
10.75 ZymoGenetics (BMS)

For more information about this report visit https://www.researchandmarkets.com/r/odq5je

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