Yet, despite the “stormy waters,” global sales of orphan drugs are projected to exceed $400 billion by 2032, according to a new report by Evaluate.

DUBLIN--(BUSINESS WIRE)--The "Orphan Drugs - Global Strategic Business Report" report has been added to ResearchAndMarkets.com's offering. The global market for Orphan Drugs was estimated at US$179.5 ...

The One Big Beautiful Bill Act takes a big step in the rare disease space by expanding the contours of the orphan drug exclusion, a once narrow exception that permitted manufacturers of rare disease ...

The One Big Beautiful Bill Act removed a number of orphan drugs from being subject to price negotiations, and this could cost the Medicare program $8.8 billion over the course of about 10 years. That ...

The 20th and 21st centuries have witnessed a profound revolution in medical science, significantly enhancing living standards and extending the life expectancy of billions worldwide. However, certain ...

The One Big Beautiful Bill Act ("OBBB") expands exemptions for orphan drugs from mandatory Medicare price negotiations and modifies their eligibility timeline. On July 4, 2025, President Trump signed ...

Policy strategist Kristi Martin discusses how the Orphan Drug Act reshapes rare disease treatment and drug-pricing challenges. In this episode, Kristi Martin, a longtime Washington, DC–based policy ...

IFx-2.0 received orphan drug designation for cutaneous melanoma, offering benefits like market exclusivity and tax credits. Phase 1 study data showed IFx-2.0 benefits patients resistant to anti-PD1 ...

The biopharma industry has hit a setback in its ambition to leverage President Donald Trump’s “big, beautiful bill” to amend an Inflation Reduction Act (IRA) provision currently affecting treatments ...

Rilzabrutinib is an investigational, oral reversible Bruton tyrosine kinase inhibitor. The Food and Drug Administration (FDA) has granted Orphan Drug designation to rilzabrutinib for the treatment of ...

The Orphan Drug designation was supported by data from an open-label phase 2 trial that included 8 adult patients with mild to moderate Behçet disease and active oral and/or genital ulcers. The Food ...