Lab scientist with safety goggles using microscope in research lab

MARKET TRENDS

16 Apr 2026

The Billion-Dollar Race to Cure the Rare

Orphan drug markets are projected to surpass $243 billion this year as genetic testing and precision medicine transform the biotech landsc...

Darzalex injection boxes displayed on pharmacy shelf

MARKET TRENDS

2 Apr 2026

Curing the Few, Billing the Many

Rare disease therapies are on track to top $400B in global sales by 2032, buoyed by policy tailwinds and a blockbuster pipeline

FDA headquarters sign with Department of Health and Human Services seals

INSIGHTS

30 Mar 2026

FDA Bets on Biology Over Bureaucracy

FDA's Plausible Mechanism Framework lets gene and RNA therapies win full approval through molecular evidence alone

U.S. FDA logo shown on mobile device screen representing drug regulation

INNOVATION

25 Mar 2026

Stem Cells Step Up for Failing Eyes

DSP-3077 wins FDA orphan status, signaling growing regulatory momentum for regenerative therapies in rare disease

Blocks labeled Rare Diseases beside stethoscope on medical desk surface

MARKET TRENDS

19 Mar 2026

Why Small Molecules Are Back in the Spotlight

Drugmakers pivot to scalable small molecule therapies in rare disease pipelines

FDA paperwork on desk with stethoscope and medication capsules

INSIGHTS

16 Mar 2026

The $100 Million Voucher Is Back From the Brink

Congress secures drug developer incentives and settles a high-profile legal battle over market exclusivity

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