News Sanofi draws up $9.1bn plan to buy Blueprint Meds Sanofi has bolstered its rare disease business with an agreement to buy Blueprint Medicines and blockbuster-in-waiting Ayvakit for ASM.
News Axe falls on Stealth Bio staff after FDA rejects lead drug Stealth BioTherapeutics' filing for Barth syndrome therapy elamipretide has been turned down by the FDA, but there may be a way forward for the drug.
News Manufacturing issues scupper Savara's lead drug filing Savara shares are pummelled after the FDA says it cannot accept a filing for lead drug Molbreevi, but it is still hopeful of an approval in 2026.
News NICE relents and backs drug for IgAN from CSL Vifor Thousands of people living with rare kidney disease IgAN in the UK will soon be able to access a new oral treatment from CSL Vifor.
News MSD adds new indication for Welireg in rare tumours MSD's oral HIF-2 alpha inhibitor Welireg has a third FDA approval, becoming the first oral treatment for a rare solid tumour PPGL or 'pheo para'.
News Abeona prices rare cell disorder gene therapy at $3.1m Abeona Therapeutics' Zevaskyn has been approved in the US as a treatment for the rare skin disorder recessive dystrophic epidermolysis bullosa (DEB).
