Regulatory analysis, drug repurposing case studies, and market intelligence — written by the team building computational tools for orphan drug discovery.
Our computational screen independently ranked the correct drug family in the top 0.3% of 1,170 candidates — without access to downstream clinical data. Here's what four independent studies found.
A failed angina drug became Viagra, then Revatio, then the most-prescribed off-label treatment in pediatric pulmonary hypertension. Three lessons from 30 years of one molecule's regulatory journey.
A regulatory pathway analysis is only useful if you understand what each section actually answers. We walk through a real OrphanRegPath report — Sirolimus repurposed for progeria — section by section.
Congress quietly reversed a five-year-old ruling that crashed orphan drug approvals by 56%. For repurposing companies, previously blocked territory just reopened — and the FDA's retroactive audit creates a narrow window of strategic value.
Patient count is a misleading metric for disease burden. When measured in life-years lost, 400 progeria patients carry the same burden as 7,800 pancreatic cancer deaths.