Delhi, May 14, 2025 (GLOBE NEWSWIRE) -- US Orphan Designated Drugs Market Opportunity, Drugs Sales, Price, Dosage and Clinical Trials Insight 2030 Report Offering and Highlights:
- US Orphan Designated Drugs Market Opportunity: > US$ 190 Billion By 2030
- Insight On FDA Designated Orphan Drugs In Clinical Trials: > 850 Orphan Drugs
- Clinical Trials Insight By Company, Indication, Phase and Priority Status
- Insight On FDA Designated Marketed Orphan Drugs: > 500 Orphan Drugs
- Pricing and Dosage Insight: > 400 Marketed Orphan Drugs
- US, Global, Regional, Annual Sales Insight (2019 – Q1’2025): >150 Orphan Drugs
- Sales, Price and Dosage Data Represented In More Than 1000 Charts and Tables
- Orphan Designation Insight By Indication, Company, Trial Phase, Marketed Drugs Represented In 1000 Tables
Download US Orphan Designated Drugs Market Opportunity, Drugs Sales, Price, Dosage and Clinical Trials Insight 2030 Report:
https://www.kuickresearch.com/report-fda-orphan-drug-database
Research Methodology:
This report on the US orphan designated drugs market is the result of comprehensive primary and secondary research, encompassing over 1400 FDA designated orphan drugs, alongside in-depth analysis of their pricing, dosing, and sales data. Market size, marketed drugs regional sales analysis and recent trends are also included in the report. To ensure the accuracy and reliability of our analysis on US orphan designated drugs pricing and market performance, we leveraged an extensive array of sources, including company reports, exchange filings, annual and quarterly reports, and official press releases.
- Over 50000 distinct web links were reviewed for comprehensive clinical trial information.
- For annual, quarterly, global and regional sales analysis, more than 1500 PDF documents were analyzed.
- More than 2000 distinct web links were examined to gather detailed drug pricing and dosage information
- More than 400 orphan designated drugs specific websites were accessed for drug profiling
- More than 2000 distinct web links were accessed to validate FDA designated orphan drug indications by indications and developer.
Throughout the world, there are numerous diseases that occur in only a few patients, and in many cases, they have limited or no treatment. For pharmaceutical companies, creating therapies for these rare diseases, which are also known as orphan diseases, has historically been economically impractical. The reason lies in the fact that the market is so minute that the return on investment hardly ever pays for the significant costs of research and development. Due to this fact, rare disease patients have long suffered from the practical difficulty of getting access to treatments specifically designed for their special conditions.
Seeing this niche, the US federal government acted on behalf of suffering patients by authorizing the passage of the Orphan Drug Act of 1983, an innovative legislation with the aim to promote the establishment of drugs to treat rare illnesses through a menu of financial incentives and regulatory perks. Since the act went into effect, the Orphan Drug Act has worked to turn once-overlooked medicine into an exciting and robust sector of the pharmaceutical market.
To date, as of May 14, 2025, the US Food and Drug Administration (FDA) has issued Orphan Drug Designation (ODD) to over 7,300 molecules and drugs. Of these, over 1,300, or about 17.9%, have come through the approval process successfully. These statistics reflect the increasing interest and activity in the orphan drug sphere. Statistically, since 2020, over half of all new drug approvals by the Center for Drug Evaluation and Research (CDER) at the FDA annually have been granted orphan status. This indicates how important orphan drugs have become in the overall strategy of treating rare and complex diseases.
The incentives provided by the Orphan Drug Act are one of the main reasons pharmaceutical firms are now more inclined to pursue treatments for orphan diseases. The incentives involve federal grants to fund clinical trials, tax credits for research costs, exemptions from some FDA fees, and quite possibly most significantly, a seven-year marketing exclusivity post-approval. This exclusivity bars competitors from bringing similar products for the same indication to market, providing a vital window of opportunity for companies to recover their investment.
A recent example of the Orphan Drug Designation at work is Thermosome's development of THE001, a thermosensitive liposomal doxorubicin formulation. THE001 has been classified as an orphan drug to treat soft tissue sarcoma, a rare form of cancer that occurs in merely 1% of all cancers affecting adults. Although the limited patient base may render uncertain the commercial prospects of the drug in regular market conditions, the orphan status works considerably in curtailing development expenses and enhancing the way to market.
While oncology is the largest of the orphan drug areas, several other disease categories are also receiving growing attention. Therapies for metabolic diseases, neurological disorders, and autoimmune or inflammatory conditions all are taking advantage of the incentives offered under the Orphan Drug Act.
An aspect that tends to attract public criticism is the hefty price tag of orphan drugs. Due to the fact that they treat very limited patient groups, and in many cases have involved intricate manufacture, these treatments are some of the costliest in the world. A recent example is Zevaskyn (prademagene zamikeracel), a gene therapy put on the market by Abeona Therapeutics for recessive dystrophic epidermolysis bullosa. Approved in April 2025, Zevaskyn has a list price of US$ 3.1 Million and is considered one of the costliest therapies to make it to market. Such exorbitant prices are usually defended on the grounds of the expense of development, the difficulty of running small-scale clinical trials, and the urgent need for effective therapies where alternatives do not exist.
Therefore, drug companies now see the orphan drug model not just as a humanitarian boon but as a sound business strategy. Fewer competitors, shorter FDA approval times, and market exclusivity have made orphan drugs one of the fastest-growing categories in the global pharmaceutical industry. Industry estimates indicate that by 2030, US orphan drugs market may surpass USD 190 Billion opportunity in annual sales.
