🧬 This Week in Rare Diseases: Global Policy Shifts, FDA Approvals, New Gene Therapies, a $20M CRISPR Push and More
From a historic WHO resolution elevating rare diseases to global health priority status to the FDA’s greenlight for the first oral on-demand treatment for HAE, this week’s roundup captures the regulatory wins, patient-led advocacy, and scientific momentum reshaping the rare disease landscape.
Whether you work in biotech strategy, clinical research, public health policy, or patient advocacy, these updates are packed with insights to keep you ahead of the curve.
🧪 In This Episode:
🌍 WHO adopts rare disease resolution with backing from Greece and 25+ countries
😢 FDA rejection of elamipretide sparks patient backlash over rare mitochondrial disease access
🇬🇧 UK charities call for systemic reform—greater than 95 percent of rare disease patients lack effective treatment
💊 FDA approves KalVista’s Ekterly—first oral, on-demand therapy for hereditary angioedema
🚀 FDA launches Complex Needs Priority Voucher to drive innovation in underserved diseases
🍁 Canada reimburses OXLUMO nationwide for PH1—tackling kidney damage across age groups
🇰🇷 Korean VHL patients demand reimbursement for $16,600/month Welireg; 50,000+ sign petition
🧬 Fujifilm’s FF-10832 earns FDA orphan status for aggressive biliary tract cancer
🧠 Mustang Bio’s MB-101 CAR-T shows durable brain cancer responses—FDA orphan designation granted
🇪🇺 EMA backs SPG601 for Fragile X syndrome following Phase 2 brain biomarker improvements
🧫 FDA designates Adcentrx’s STEAP1-targeting gastric cancer therapy ADRX-0405 as orphan drug
❤️ Solid Bio’s SGT-501 enters clinical trial phase for CPVT—targeting calcium regulation to prevent arrhythmias
🧒 $20M CRISPR center launched by Chan Zuckerberg Initiative to scale custom pediatric rare disease cures
Whether you’re tracking clinical milestones, monitoring regulatory shifts, or exploring cutting-edge therapeutics, this episode delivers the pulse of global rare disease innovation.
📢 Stay Ahead in Rare Disease Research!
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