Do rare disease drugs with tiny patient populations lie outside the profitability continuum that runs from small-molecule primary care products to specialist biologics?

Amarin’s drug, which costs £144.21 for a 30-day supply, is to be made available on the National Health Service in England and Wales for reducing the risk of cardiovascular events after NICE reversed its previous rejection of the product.

The biomanufacturing firm National Resilience has raised more than $2bn in venture capital and millions more in fees from government and industry contracts since 2020. Also, Code Biotherapeutics raised $75m in series A cash, Concert grossed $66.4m and Amarin is cutting US jobs. 

Roche is paying $125m upfront to in-license potential blockbuster lead candidate camonsertib from Repare, which calls it a best-in-class ATR inhibitor and has the chance to opt back in on US commercialization.