Despite a sceptical appraisal by FDA reviewers, Alnylam's Onpattro has been recommended for approval as a treatment for transthyretin amyloidosis cardiomyopathy (ATTR-CM)
BridgeBio is getting ready to file for FDA approval of its acoramidis drug for transthyretin amyloidosis (ATTR) cardiomyopathy, armed with positive results in a phase 3 tr
Pfizer reckons it will make a whopping $22 billion in sales of its oral COVID-19 antiviral Paxlovid in 2022, with another $32 billion from its BioNTech-partnered coronavirus vaccine Comirna
AstraZeneca has clearly made the rare disease transthyretin amyloidosis (ATTR) a key component of its rare disease pipeline, licensing a second drug candidate in the space of a couple of mo
NICE has rejected regular NHS funding for Pfizer’s Vyndaqel (tafamidis) for a rare heart condition, although it has left the door open for negotiations if the pharma decid
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