With the safety of gene therapies thrown into the spotlight by a series of FDA clinical holds, Astellas has said that an interim readout in a phase 1/2 trial of its Pompe disease candidate
Sanofi has won FDA approval for its sutimlimab for cold agglutinin disease (CAD) at the second time of asking, becoming the first approved therapy for the rare blood disorder in the US.
In this second article of a three-part series exploring pricing in Europe, CRA’s Life Sciences Practice team discusses the role that disease rarity plays in reimbursement decisions among pa
NHS England has agreed a confidential deal with Orchard Therapeutics that will allow access to Libmeldy – a gene therapy for a rare childhood disease that is thought to be the most expensiv
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
After regulatory hold-ups in the US and Europe, Ipsen has claimed a confidence-boosting approval in Canada for palovarotene, a rare disease therapy acquired via its $1.3 billion takeover of