Dutch biotech Pharming has claimed FDA approval for Joenja, the first drug indicated for recently identified rare immunodeficiency disorder activated phosphoinositide 3-ki
Pharming has submitted its oral phosphoinositide 3-kinase delta (PI3Kδ) inhibitor leniolisib for approval in the EU as a treatment for activated PI3K delta syndrome (APDS), an ultra-rare di
Australia's CSL has said its one-monthly treatment for hereditary angioedema has shown it is safe and effective in a phase 3 trial, and will be filed for approval in its current financial y
Pharming has taken a step closer to getting a second product to market, after its rare disease therapy leniolisib met its objectives in a pivotal trial, setting up regulatory filings in the
The US FDA announced today that it has cleared two novel gene therapies for sickle cell disease: Vertex Pharmaceuticals' Casgevy and bluebird bio's Lyfgenia.
On 1st October, the roll-out of the National Contract Value Review (NCVR) process marked a pivotal moment in the trajectory of clinical research in the UK, introducing a standardised, natio