FDA Designated US Orphan Drugs Rare Disease Drug Sales Market Size Clinical Trial Insight
US Rare Disease Drug Sales Market Opportunity To Surpass USD 190 Billion By 2030 Says Kuick Research
Delhi, Oct. 16, 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 Report: https://www.kuickresearch.com/report-fda-orphan-drug-database
The Orphan Drug Designation program of the FDA is a significant element in the development of therapies for rare diseases, offering great opportunities in the orphan drug market. The US orphan drug market is anticipated to reach more than US$ 190 Billion by the year 2030, driven by increased demand for therapies aimed at treating rare and complicated diseases. This market is motivated by regulatory incentives as well as by the possibility of high margins because the patient populations for these diseases are relatively small.
More than 850 FDA designated orphan drugs are in clinical trials today in the US, reflecting the high interest in unmet medical needs in areas of rare disease. The orphan drug clinical trial market has many companies targeting various indications, ranging from genetic diseases to orphan cancers. The trial stages are varied, with a number of therapies progressing into later-stage trials and others remaining in initial stages of development. FDA-designated orphan drugs receive priority review status, accelerating their path from laboratory to patient availability.
To date, over 500 FDA designated orphan drugs have been successfully approved for use in clinical settings in the US, with hundreds more in development. This growing portfolio highlights the potential for growth and profitability in the orphan drug market. Pricing and dosage information for more than 400 commercialized orphan drugs gives stakeholders valuable information on navigating this specialty. Knowledge of the differences in drug pricing, dosing regimens, and overall market realities is vital for companies and investors as they determine the viability of orphan drugs in their product portfolios. The US orphan drug market has indicated robust sales patterns, with our report recording over 150 orphan drugs which have been assessed for yearly sales ranging from 2019 to the first quarter of 2025. These estimates, quantified in over 1,000 tables and charts, present a full picture of the financial pattern of orphan drug sales, price ranges, and dosing habits.
Recent news provides examples of the increasing application of orphan drugs in trials. Transpire Bio in October 2025 was given Orphan Drug Designation by the FDA for two novel treatments: TRB-ILD1 for Idiopathic Pulmonary Fibrosis (IPF) and TRB-ONC1 for glioblastoma (GBM). These designations provide development incentives to Transpire Bio that can expedite the development of these treatments. Another example is Minovia Therapeutics, which in mid-October 2025 was granted Orphan Drug Designation for MNV-201 for the treatment of myelodysplastic syndrome (MDS). This in addition to its Fast Track status represents an important milestone in helping the advancement of treatments for rare hematologic conditions.
All in all, the FDA’s Orphan Drug Designation program remains a driving force for innovation, generating a high-powered market that is poised for strong growth in the future.
Neeraj Chawla Research Head Kuick Research neeraj@kuickresearch.com +911147067990 https://www.kuickresearch.com/
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