Rare Disease Drug Approvals

Oct 02, 2026

Summary  Ataxia-telangiectasia is a rare, progressive genetic disorder affecting an estimated 1 in 40,000–100,000 people worldwide. AQNEURSA received FDA approval on September 18, 2026, becoming the first and only approved treatment for ataxia in patients with ataxia-telangiectasia and marking the first...

Sep 07, 2026

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...