PASATRU
Sep 07, 2026
Rare Disease Drug Approvals: Why Are Ultra-Orphan Therapies Suddenly Big Pharma’s Favorite Gamble?
Summary Diseases once dismissed as “too rare to matter” (hundreds to low‑thousands of patients globally) are now central to innovation, with regulators, payers, and major companies adapting fast. In the first eight months of 2026, the FDA cleared more than 6 first‑in‑class drugs, a pace that historically too...
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