DUBLIN–(BUSINESS WIRE)–The «U.S. Orphan Designated Drugs Market Opportunity, Drugs Sales, Price, Dosage & Clinical Trials Insight 2030» report has been added to ResearchAndMarkets.com’s offering.
Report Offering & Highlights:
- U.S. Orphan Designated Drugs Market Opportunity: > U.S.$ 190 Billion by 2030
- Insight on FDA Designated Orphan Drugs in Clinical Trials: > 850 Orphan Drugs
- Clinical Trials Insight by Company, Indication, Phase & Priority Status
- Insight on FDA Designated Marketed Orphan Drugs: > 500 Orphan Drugs
- Pricing & Dosage Insight: > 400 Marketed Orphan Drugs
- U.S., Global, Regional, Annual Sales Insight (2019 – Q1’2025): >150 Orphan Drugs
- Sales, Price & Dosage Data Represented in More Than 1000 Charts & Tables
- Orphan Designation Insight by Indication, Company, Trial Phase, Marketed Drugs Represented in 1000 Tables
In the future, the U.S. market for orphan drugs will continue to grow. The regulatory environment will continue to shift to facilitate orphan drug development, with increasingly streamlined approval procedures and additional incentives to stimulate innovation. As rare diseases are increasingly well understood, and as advances in science offer new means of attacking them, the market for orphan drugs will increasingly be characterized by diversity in the types of diseases being treated. There is also increased awareness that rare diseases tend to have common molecular mechanisms, raising the possibility that treatments applicable across larger groups could be developed.
In addition, the development of the orphan drug market has contributed to the rise in collaborations between pharmaceutical firms. Collaborations can assist in the division of the cost and the acceleration of the development of new medicines. An example is the partnership between Cadrenal Therapeutics and Abbott, established in March 2025 to aid in the development of tecarfarin, a new oral anticoagulant, in patients with HeartMate 3T Left Ventricular Assist Devices (LVADs).
Tecarfarin, granted orphan drug status, is under investigation in a clinical trial for enhancing anticoagulation benefit in patients with advanced heart failure. This alliance reflects the increased trend of grouping together resources and expertise to make therapies for rare conditions, as the scientific and logistical hurdles associated with orphan drug development can be significant.
More drugs have over the years acquired orphan drug status. To date, hundreds of drugs in development have been assigned such status with the indications ranging from rare tumors to genetic conditions, cardiovascular diseases, and neurologic conditions. The FDA orphan drug designation not only encouraged the development of novel treatments but also has resulted in some unexpected commercial success stories. Those drugs initially designed for limited patient populations now are showing to be profitable products, reversing the opinion that orphan drugs cannot exhibit good market performance.
One of the best examples is Merck’s Keytruda (pembrolizumab), an immune checkpoint inhibitor that originally received orphan drug designation in 2012 for malignant melanoma. Since then, Keytruda has had its indications broadened to include various cancers, such as esophageal carcinoma, lymphoma, and lung cancer, among others, many of which are orphan diseases. Although an orphan drug, Keytruda is now amongst the world’s best-selling cancer medications, with 2024 revenue of almost U.S.$ 29.4 billion, an astonishing 18% increase over last year. Importantly, more than 60% of this originated from the U.S. alone. This business success demonstrates how orphan drugs, in the long run, can discover larger markets and generate sizeable returns.
Likewise, Gilead’s Biktarvy, an HIV drug, was given orphan drug designation and is now one of the company’s best-selling drugs. In 2024, it recorded U.S.$ 13.4 billion in sales, up 13% from the previous year. Both drugs underscore how treatments for rare diseases can evolve from specialty therapies into big moneymakers.
The success of these drugs in the market shows that orphan drug development can be both clinically effective and profitable. With appropriate incentives, companies are more likely to invest in the treatment of rare diseases, even if the number of patients is small. The incentives created by the FDA and other regulatory authorities, including the potential for market exclusivity and fast-track review, enable firms to break even on their investment and reap huge profits in the long term. These advantages have motivated numerous firms to enter the orphan drug area, particularly since rare diseases are increasingly being understood as a result of advances in genomics and biotechnology.
Key Topics Covered:
1. FDA Orphan Designation Criteria & Market Exclusivity
2. US Orphan Designated Drugs Market Insight
2.1 Current Market Overview
2.2 Future Market Opportunity Assessment
3. US Orphan Designated Drugs Market Trends by Indication
3.1 Cancer
3.2 Neurological Disorders
3.3 Cardiovascular Disorders
3.4 Ophthalmic Diseases
3.5 Rare Genetic Disorders & Metabolic Disorders
3.6 Autoimmune & Inflammatory Diseases
4. US Orphan Designated Drugs Reimbursement Scenario
4.1 Medicare
4.2 Medicaid
4.3 Private Insurers
4.4 Pharmaceutical Companies
5. US Orphan Designated Drugs Clinical Trials Insight
5.1 by Patient Segment
5.2 by Phase
5.3 by Priority Status
6. US Orphan Designated Drugs Clinical Trials by Company, Indication & Phase
6.1 Research
6.2 Preclinical
6.3 Phase-I
6.4 Phase-I/II
6.5 Phase-II
6.6 Phase-II/III
6.7 Phase-III
6.8 Preregistration
6.9 Registered
7. Marketed Orphan Designated Drugs Clinical Insight by Company & Indication
8. US Orphan Designated Drugs Dosage, Price & Treatment Cost Insight
8.1 Lynparza
8.2 Nplate
8.3 Tafinlar
8.4 Halaven
8.5 Jadenu
8.6 Xpovio
8.427 Ojemda
8.428 Ojjaara
8.429 Piasky
8.430 Rezurock
8.431 Pombiliti
8.432 Ogsiveo
8.433 Qfitlia
8.434 Zevaskyn
9. US Orphan Designated Drugs Sales Insight (2019 – Q1’2025)
9.1 Amvuttra
9.2 Krazati
9.3 Tecvayli
9.4 Imjudo
9.5 Rezurock
9.6 Elrexfio
9.7 Fabhalta
9.154 Xospata
9.155 Defitelio
9.156 ReFacto
9.157 Praluent
9.158 Mozobil
9.159 Yescarta
9.160 Lunsumio
9.161 Keytruda
9.162 Pemazyre
9.163 Camzyos
10. Competitive Landscape
10.1 AbbVie
10.2 Alexion AstraZeneca Rare Disease
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 BioMarin Pharmaceutical
10.11 Bioverativ
10.12 Boehringer Ingelheim
10.13 Bristol-Myers Squibb
10.14 Chiesi
10.15 Chugai Pharmaceutical
10.16 Collaborations Pharmaceuticals
10.17 CSL
10.18 Daiichi Sankyo Company
10.19 Eisai Co Ltd
10.20 Eli Lilly & Company
10.21 Emergent BioSolutions
10.22 Ferring Pharmaceuticals
10.23 Flavocure Biotech
10.24 Genentech
10.25 Genmab
10.26 Gilead Sciences
10.27 GSK
10.28 Hanmi Pharmaceutical
10.29 Horizon Therapeutics plc
10.30 Incyte Corporation
10.31 Ionis Pharmaceuticals
10.32 Ipsen
10.33 Janssen Biotech
10.34 Jazz Pharmaceuticals plc
10.35 Johnson & Johnson
10.36 Kamada
10.37 Krystal Biotech
10.38 Kyowa Kirin
10.39 Leadiant Biosciences
10.40 Ligand Pharmaceuticals
10.41 Lundbeck A/S
10.42 MeiraGTx
10.43 Merck
10.44 Moderna Therapeutics
10.45 Novartis
10.46 Novo Nordisk
10.47 Omeros Corporation
10.48 Onyx Pharmaceuticals (Amgen)
10.49 Otsuka Pharmaceutical
10.50 Pfizer
10.51 PTC Therapeutics
10.52 Recordati
10.53 Regeneron Pharmaceuticals
10.54 Roche
10.55 Sanofi
10.56 Servier
10.57 Shionogi
10.58 Spark Therapeutics
10.59 Sumitomo Pharma
10.60 Sutro Biopharma
10.61 Swedish Orphan Biovitrum
10.62 Takeda
10.63 Teva Pharmaceutical Industries
10.64 UCB
10.65 UniQure
10.66 Vertex Pharmaceuticals
10.67 XOMA
10.68 ZymoGenetics (BMS)
For more information about this report visit https://www.researchandmarkets.com/r/o8c6f1
About ResearchAndMarkets.com
ResearchAndMarkets.com is the world’s leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.
Contacts
ResearchAndMarkets.com
Laura Wood, Senior Press Manager
[email protected]
For E.S.T Office Hours Call 1-917-300-0470
For U.S./ CAN Toll Free Call 1-800-526-8630
For GMT Office Hours Call +353-1-416-8900
