FDA released its 2026 Rare Disease Hub Strategic Agenda and a draft guidance on 3-year New Clinical Investigation Exclusivity. The agency also outlined new pathways including the plausible mechanism pathway and Rare Disease Evidence Principles to accelerate treatments for rare diseases.
Fujifilm Biotechnologies has opened the UK's largest single-use biopharmaceutical CDMO facility in Teesside with a £400 million investment. The 110,000 sq ft facility features 19,000 liters of bioreactor capacity and will be operational in H1 2026. The expansion includes a new Bioprocess Innovation Centre that doubles the site's lab footprint.
The FDA has established a "plausible mechanism pathway" to approve personalized genome editing and RNA-based therapies for rare and ultra-rare diseases without requiring large randomized controlled trials, streamlining access to individualized treatments.
The U.S. orphan drug market is projected to exceed $200 billion by 2031, driven by innovation in oncology and genetic disorders. More than 1,400 FDA-designated orphan drugs are in development or marketed, with over 800 currently in clinical trials.
