A stark transatlantic regulatory divide isolates patients in one hemisphere from life-changing orphan therapies.
The growing regulatory and philosophical division between the FDA and the EMA over historical data validity and trial endpoints splits the market, stranding patients with rare diseases without approved treatments in one region.
The same conclusion keeps arriving from across the workspace's research — 1 topics independently instantiate this theme. Filter the evidence by where it came from:
European regulators refused approval for the rare-disease therapy, preventing patients in Europe from accessing a drug already cleared in the US.
Despite evaluating the same clinical package, the EMA's rejection of the rare disease drug narsoplimab prevents European patients from accessing a therapy already approved in the US.
European approvals based on a landmark neuroinflammation trial have not been matched in the U.S., stalling patient access to the first disease-progression multiple sclerosis therapy.
The European rejection of narsoplimab prevents European TA-TMA patients from receiving a life-saving orphan therapy already approved in the US.
The EMA's refusal to approve narsoplimab, despite FDA's prior approval, leaves European TA-TMA patients without access to the only approved therapy for this rare disease.