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

Novartis logo displayed on corporate building exterior

PARTNERSHIPS

10 Mar 2026

Why Novartis Just Spent $12B on a Tiny Biotech

Novartis completes $12B Avidity acquisition, gaining RNA muscle-targeting platform and three rare neuromuscular therapies

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