Posts

AstraZeneca foresees blockbuster sales for Claudin 18.2 ADC, but Ultomiris flops in rare disease

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AstraZeneca’s pipeline struggles continue with disappointing late-stage cancer and rare disease data

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In another reversal for rare disease field, FDA to reconsider Regenxbio gene therapy

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Busy FDA gives rare disease sector complementary pathways, unanswered questions – 2026 snapshot

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Lilly signs up to $1.26B deal for Hanmi's GLP‑2 drug sonefpeglutide for short bowel syndrome and other indications

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BioMarin's rare disease therapy shows no clinical benefit in Phase 3 test

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Biogen's Post-Apellis Strategy: Expanding Early-Stage Pipeline to Drive Next-Generation Growth

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Chiesi Group Acquires KalVista Pharmaceuticals for $1.9 Billion to Expand Rare Disease Portfolio

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FDA Grants Accelerated Approval to Denali's Avlayah for Hunter Syndrome, Defying Rare Disease Rejection Trend

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Inovio Trims Team as Trouble Brews at FDA for Rare Disease Candidate

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FDA Accelerates Rare Disease Drug Approvals with New Frameworks and Record 2025 Approvals

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Senate Hearing Criticizes FDA's Rare Disease Drug Review Process

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Ultragenyx Announces Layoff of 130 Employees Amid FDA Delays on UX111 Resubmission

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Amgen Rejects FDA Request to Withdraw Rare Disease Drug Tavneos

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BioMarin Veterans Launch Mendra Biotech with $82M for AI-Driven Rare Disease Drugs

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BioMarin Veterans Launch Mendra Biotech with $82M for AI-Driven Rare Disease Drugs

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TriNetX Achieves Record as the Most Cited Real-World Electronic Health Record Dataset in Peer-Reviewed Research

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Unlocking Japan's Potential in Rare Disease Drug Development: Recent Progress and Challenges (2025)

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Alkermes Acquires Avadel Pharmaceuticals for $2.1B, Gaining Sleep Drug LUMRYZ and Setting Strategic Evolution

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Sanofi’s efdoralprin alfa (INBRX-101) achieves all endpoints in Phase 2 alpha-1 antitrypsin deficiency (AATD) trial

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