The difficulty of finding and retaining patients with orphan diseases translates into higher marketing costs and adds to other restrictions that limit the attractiveness of some orphan drugs. Will the IRA exacerbate matters?

Efficacy in PROpel study was driven by the small percentage of patients with tumor BRCA mutations, agency says, raising concerns that olaparib may amount to a ‘toxic placebo’ in those without the mutation; Oncology Drugs Advisory Committee will consider whether new indication in metastatic castration-resistant prostate cancer should be restricted by mutation status.

The Swiss company has raised its 2023 forecast thanks to stronger Q1 sales, but its transformation towards a more ‘pure-play’ pharma company is still a work in progress.

Public Company Edition: ImmunoGen received the first $75m under a $175m term loan, while Protagonist raised $100m in a follow-on offering, but companies with bankruptcy filings and job cuts outpaced those accessing new capital by a wide margin in late March and early April.